regulatory

AbbVie Submits Regulatory Applications to the US FDA and EMA for Rinvoq (upadacitinib) to Treat Active Ulcerative Colitis

Shots: The application is based on 2 P-III U-ACHIEVE & U-ACCOMPLISH induction studies & 1 U-ACHIEVE maintenance study evaluating upadacitinib (45mg, qd) as IT & (15/30 mg, qd) as MT vs PBO in patients with mod. to sev. UC The studies met its 1EPs i.e., patients achieved clinical remission (per Adapted Mayo Score) & 2EPs …

AbbVie Submits Regulatory Applications to the US FDA and EMA for Rinvoq (upadacitinib) to Treat Active Ulcerative Colitis Read More »

BioCryst’s Orladeyo (berotralstat) Receives NICE Recommendation as First Oral Therapy to Prevent HAE Attacks in the UK

Shots: The NICE recommendation is based on P-III APeX-2 trial evaluating Orladeyo vs PBO in adult & pediatric patients aged ≥12yrs. with HAE attacks The trial met its 1EPs i.e., reduction in HAE attacks @24wks. & sustained through 96wks. with an 80% reduction in patients with mean HAE attack rate/mos. @ 25-96wks. The therapy was …

BioCryst’s Orladeyo (berotralstat) Receives NICE Recommendation as First Oral Therapy to Prevent HAE Attacks in the UK Read More »

BeiGene’s Brukinsa (zanubrutinib) Receives the US FDA’s Accelerated Approval for the Treatment of R/R Marginal Zone Lymphoma

Shots: The approval is based on 2 trials i.e., P-II MAGNOLIA & P-I/II BGB-3111-AU-003 trial evaluate Brukinsa (160mg, bid or 320 mg, qd) in 66 & 20 patients with R/R MZL who received at least 1 anti-CD20-based regimen Both trial demonstrated ORR (56% & 80%), CR rate (20% & 20%) based on assessment using CT …

BeiGene’s Brukinsa (zanubrutinib) Receives the US FDA’s Accelerated Approval for the Treatment of R/R Marginal Zone Lymphoma Read More »

Amgen’s Lumakras (sotorasib) Receives Health Canada’s Approval for KRAS G12C-Mutated Locally Advanced or Metastatic Non-Small Cell Lung Cancer

Shots: The approval is based on P-II CodeBreaK 100 trial evaluating the efficacy and tolerability of Lumakras (PO, qd) in 126 patients with KRAS G12C mutation-positive advanced NSCLC whose disease had progressed after prior treatment with immunotherapy or CT The 1EPs were centrally assessed ORR. Additionally, the therapy demonstrated a positive benefit-risk profile with rapid, …

Amgen’s Lumakras (sotorasib) Receives Health Canada’s Approval for KRAS G12C-Mutated Locally Advanced or Metastatic Non-Small Cell Lung Cancer Read More »

Hutchmed’s Amdizalisib (HMPL-689) Receives Breakthrough Therapy Designation for the Treatment of R/R Follicular Lymphoma in China

Shots: The NMPA has granted BTD to amdizalisib for the treatment of r/r FL The preliminary results from the ongoing P-Ib expansion study showed that the therapy was well- tolerated, exhibit dose-proportional PK, manageable toxicity profile, clinical activity in r/r B-cell lymphoma & results will be presented at ESMO 2021. Currently, the company gains all …

Hutchmed’s Amdizalisib (HMPL-689) Receives Breakthrough Therapy Designation for the Treatment of R/R Follicular Lymphoma in China Read More »

BeiGene and Novartis Report the US FDA’s Acceptance of BLA for Tislelizumab to Treat Esophageal Squamous Cell Carcinoma

Shots: The BLA submission is based on a P-III RATIONALE 302 trial evaluating the efficacy and safety of tislelizumab vs CT in 1972 patients with advanced or metastatic ESCC who had received prior systemic therapy. The anticipated PDUFA date for the therapy is July 12, 2022 The trial met its 1EPs i.e., the therapy showed …

BeiGene and Novartis Report the US FDA’s Acceptance of BLA for Tislelizumab to Treat Esophageal Squamous Cell Carcinoma Read More »

Boehringer Ingelheim and Eli Lilly’s Jardiance (empagliflozin) Receive the US FDA’s Breakthrough Therapy Designation for the Treatment of Heart Failure with Preserved Ejection Fraction

Shots: The designation is based on the P-III EMPEROR-Preserved trial evaluates the safety and efficacy of empagliflozin (10mg, qd) vs PBO in 5,988 patients with chronic HFpEF with/out diabetes The results demonstrated a 21% reduction in risk for the composite 1EPs of CV death or hospitalization for adults with HF. The results were presented at …

Boehringer Ingelheim and Eli Lilly’s Jardiance (empagliflozin) Receive the US FDA’s Breakthrough Therapy Designation for the Treatment of Heart Failure with Preserved Ejection Fraction Read More »

MHRA cuts could affect UK regulatory decisions, say unions

A 20% to 25% reduction in staffing at the UK Medicines and Healthcare products Regulatory Agency (MHRA) risks undermining the ability of the authority to fulfil its role, according to trade unions. In an open letter to Health Secretary Sajid Javid, Prospect, Unite and three other unions say the decision to slash the MHRA’s current …

MHRA cuts could affect UK regulatory decisions, say unions Read More »

Impel NeuroPharma’s Trudhesa (dihydroergotamine mesylate) Nasal Spray Receives the US FDA’s Approval for the Treatment of Migraine

Shots: The approval is based on the P-III STOP 301 trial evaluates Trudhesa (0.725 mg/spray) in 5650+ patients with migraine with or without aura for 24 or 52 wks. The exploratory efficacy results showed that nasal spray provides rapid, sustained, and consistent symptom relief. The therapy is well-tolerated & no serious TEAEs were observed Trudhesa …

Impel NeuroPharma’s Trudhesa (dihydroergotamine mesylate) Nasal Spray Receives the US FDA’s Approval for the Treatment of Migraine Read More »

JW’s Relmacabtagene Autoleucel Receives the NMPA’s Approval for the Treatment of R/R Large B-Cell Lymphoma in China

Shots: The approval is based on a RELIANCE study that evaluates efficacy & safety of relma-cel in 59 patients with r/r LBCL who had failed at least 2L of therapy, including CD20 agent & anthracycline & patients continue to be monitored for 2+ yrs. The results showed the high rates of durable disease response & …

JW’s Relmacabtagene Autoleucel Receives the NMPA’s Approval for the Treatment of R/R Large B-Cell Lymphoma in China Read More »

Moderna’s Spikevax Receives TGA’s Provisional Approval to Treat COVID-19 in Patients Aged 12-17 Years

Shots: TGA has provisionally approved the use of Moderna’s Spikevax (elasomeran) in individuals aged ≥12yrs. The decision follows the provisional approval granted by the TGA to Spikevax on Aug 09, 2021 for use in individuals aged ≥18yrs. Provisional approval for this group follows the data supporting the efficacy & safety of the vaccine. The recommended …

Moderna’s Spikevax Receives TGA’s Provisional Approval to Treat COVID-19 in Patients Aged 12-17 Years Read More »

Novartis Cosentyx (secukinumab) Receives NICE Recommendation for Patients with Plaque Psoriasis

Shots: NICE has recommended Cosentyx as a cost-effective option for the treatment of mod. to sev. PsO in patients aged 6-17 yrs. who have not responded to other forms of systemic treatment & for whom these options were contraindicated or not tolerated Additionally, patients will now have access to this treatment option on the NHS. …

Novartis Cosentyx (secukinumab) Receives NICE Recommendation for Patients with Plaque Psoriasis Read More »

Alexion’s Ultomiris (ravulizumab) Receives EC’s Approval for Expanded Use to Treat Paroxysmal Nocturnal Haemoglobinuria

Shots: The approval is based on the P-III trial that evaluates Ultomiris (q4w/q8w) vs Soliris in children & adolescents aged ≤18yrs. with PNH The interim result showed that the therapy was effective in achieving C5 complement inhibition @26wks. & demonstrated safety & efficacy profile with reduced dosing frequency, no treatment-related SAEs & no patients discontinued …

Alexion’s Ultomiris (ravulizumab) Receives EC’s Approval for Expanded Use to Treat Paroxysmal Nocturnal Haemoglobinuria Read More »

Janssen’s Invega Hafyera (paliperidone palmitate) Receives the US FDA’s Approval for the Treatment of Schizophrenia

Shots: The approval is based on a P-III study that evaluates Invega hafyera (q6mos., twice-yearly injectable) vs Invega trinza in 702 adults in a ratio (2:1) aged 18-70yrs. with schizophrenia from 20 countries The results showed non-inferiority on 1EPs of time to 1st relapse @12mos. in ITT & per-protocol analysis sets., patients were relapse-free (92.5% …

Janssen’s Invega Hafyera (paliperidone palmitate) Receives the US FDA’s Approval for the Treatment of Schizophrenia Read More »

BeiGene’s Brukinsa (zanubrutinib) Receives the US FDA’s Approval for the Treatment of Waldenström’s Macroglobulinemia

Shots: The approval is based on P-III ASPEN trial evaluating Brukinsa (160mg, bid, or 320 mg, qd) vs ibrutinib in 201 patients with WM who harbor an MYD88 mutation The 1EPs of the trial was VGPR rate in the overall ITT population as assessed by IRC. VGPR rate (28% vs 19%) based on the modified …

BeiGene’s Brukinsa (zanubrutinib) Receives the US FDA’s Approval for the Treatment of Waldenström’s Macroglobulinemia Read More »

Merck’s Keytruda (pembrolizumab) Receives the US FDA’s Approval for Label Update to Treat Advanced Urothelial Carcinoma

Shots: The US FDA has approved Merck’s Keytruda for the treatment of patients with locally advanced or mUC who are not eligible for any Pt-containing CT & converted the indication from an accelerated to a full approval along with a label update Previously, the FDA’s ODAC voted 5-3 in favor of maintaining the accelerated approval …

Merck’s Keytruda (pembrolizumab) Receives the US FDA’s Approval for Label Update to Treat Advanced Urothelial Carcinoma Read More »

Ascendis’ Skytrofa (lonapegsomatropin-tcgd) Receives the US FDA’s Approval for Pediatric Growth Hormone Deficiency

Shots: The approval is based on the P-III heiGHt trial evaluating Skytrofa (qw) vs somatropin in 161 treatment-naïve children ≥aged 1yrs. with GHD who have growth failure due to inadequate secretion of endogenous GH The study met its 1EPs i.e., non-inferiority in AHV & showed a higher AHV @52wks., no serious AEs or discontinuations related …

Ascendis’ Skytrofa (lonapegsomatropin-tcgd) Receives the US FDA’s Approval for Pediatric Growth Hormone Deficiency Read More »

MorphoSys and Incyte’s Minjuvi (tafasitamab) + Lenalidomide Receive EC’s Approval for the Treatment of R/R DLBCL

Shots: The conditional approval is based on the P-II L-MIND study evaluating the safety and efficacy of tafasitamab + lenalidomide in patients with r/r DLBCL who are not eligible for ASCT Results: ORR (56.8%); CR rate (39.5%); PR rate (17.3%) as assessed by an IRC; m-DoR (43.9mos.) after a minimum follow up of 35mos. Tafasitamab …

MorphoSys and Incyte’s Minjuvi (tafasitamab) + Lenalidomide Receive EC’s Approval for the Treatment of R/R DLBCL Read More »

Pfizer and BioNTech Initiate Rolling Submission of sBLA for the US FDA’s Approval of Comirnaty’s Booster Dose in Individuals Aged 16 Years and Older

Shots: The sBLA is based on P-III trial for Comirnaty (third booster dose) administered b/w 4.8 – 8mos., following the completion of 2 dose in 306 participants aged b/w 18-55 yrs. The P-III trial showed that third booster dose induces robust neutralizing Abs against wild-type strain @1mos., following PD3 with a median follow-up time of …

Pfizer and BioNTech Initiate Rolling Submission of sBLA for the US FDA’s Approval of Comirnaty’s Booster Dose in Individuals Aged 16 Years and Older Read More »

AstraZeneca’s Forxiga (dapagliflozin) Receives MHLW’s Approval for the Treatment of Chronic Kidney Disease in Patients with and without T2D

Shots: The approval is based on P-III DAPA-CKD trial evaluate Forxiga (10mg, qd) + SoC vs PBO in 4304 patients with CKD Stage 2-4 & elevated urinary albumin excretion with/out T2D The results showed a 39% reduction in risk of worsening of renal function, ESKD, CV, or renal death & 31% from any cause. Safety …

AstraZeneca’s Forxiga (dapagliflozin) Receives MHLW’s Approval for the Treatment of Chronic Kidney Disease in Patients with and without T2D Read More »

Thermo Fisher’s Oncomine Dx Target Test Receives the US FDA’s Approval as a CDx for the Treatment of IDH1-Mutated Cholangiocarcinoma

Shots: The FDA has approved the Oncomine Dx target test as a CDx to identify patients with IDH1 mutated CCA who may be candidates for Servier’s Tibsovo Oncomine Dx Target Test is NGS based CDx test that provides robust results in the IDH1 gene clinically associated with CCA & is currently approved in 15+ countries, …

Thermo Fisher’s Oncomine Dx Target Test Receives the US FDA’s Approval as a CDx for the Treatment of IDH1-Mutated Cholangiocarcinoma Read More »

Cara and Vifor’s Korsuva (difelikefalin) Receive the US FDA’s Approval for the Treatment of Moderate-to-Severe Pruritus Due to CKD in Patients with Hemodialysis

Shots: The approval is based on the two P-III trials i.e., KALM-1, KALM-2 along with additional 32 clinical studies that evaluate Korsuva vs PBO in patients with a mod. to sev. pruritus associated with CKD who undergoes hemodialysis Results: patients achieved four-point improvement from baseline on a measure of severe itch (40% & 37%) vs …

Cara and Vifor’s Korsuva (difelikefalin) Receive the US FDA’s Approval for the Treatment of Moderate-to-Severe Pruritus Due to CKD in Patients with Hemodialysis Read More »

Argenx Reports EMA’s Validation of MAA for Efgartigimod to Treat Generalized Myasthenia Gravis

Shots: The MAA is based on the P-III ADAPT trial evaluating the safety and efficacy of efgartigimod vs PBO in a ratio (1:1) in 167 adult patients with gMG for 26wks. The 1EPs was the proportion of AChR-Ab+ patients who achieved a response on the MG-ADL score defined by at least a two-point improvement for …

Argenx Reports EMA’s Validation of MAA for Efgartigimod to Treat Generalized Myasthenia Gravis Read More »

Takeda and Ono’s Opdivo (nivolumab) + Cabometyx (cabozantinib) Receive MHLW’s Approval for the Treatment of Unresectable or Metastatic Renal Cell Carcinoma

Shots: The approval is based on P-III CheckMate -9ER study evaluating ONO’s Opdivo (240 mg, IV, q2w) + Takeda’s Cabometyx (40mg, PO, qd) vs sunitinib (50mg, PO, qd, for 4wks.) in a ratio (1:1) in patients with previously untreated advanced/mRCC Opdivo + Cabometyx demonstrated an improvement in 1EPs of PFS & 2EPs of OS & …

Takeda and Ono’s Opdivo (nivolumab) + Cabometyx (cabozantinib) Receive MHLW’s Approval for the Treatment of Unresectable or Metastatic Renal Cell Carcinoma Read More »

AbbVie’s Rinvoq (upadacitinib) Receives EC’s Approval for the Treatment of Moderate to Severe Atopic Dermatitis

Shots: The approval is based on 3 P-III studies i.e., Measure Up 1/2/AD Up evaluating the efficacy & safety of Rinvoq (15/30 mg, qd) as monothx. with TCS vs PBO in 2500+ adults & adolescents with AD The studies met its 1EPs & 2EPs @16wks. i.e., improvement in skin clearance & itch reduction, patients achieved …

AbbVie’s Rinvoq (upadacitinib) Receives EC’s Approval for the Treatment of Moderate to Severe Atopic Dermatitis Read More »

UCB’s Bimzelx (bimekizumab) Receives EC’s Approval for the Treatment of Moderate to Severe Plaque Psoriasis

Shots: The approval is based on 3 P-III studies i.e., BE VIVID, BE READY, BE SURE evaluating the efficacy & safety of bimekizumab vs PBO + ustekinumab, PBO, adalimumab in 1480 patients with a mod. to sev. PsO. The EC’s approval is valid in 27 member states of the EU, Iceland, Liechtenstein & Norway The …

UCB’s Bimzelx (bimekizumab) Receives EC’s Approval for the Treatment of Moderate to Severe Plaque Psoriasis Read More »

Pfizer and BioNTech’s Comirnaty Receive the US FDA’s Approval to Prevent COVID-19 in Individuals Aged 16 Years and Older

Shots: The approval is based on the longer-term follow-up data from the P-III trial that showed high efficacy and favorable safety profile through 6mos. after the second dose The vaccine will now be marketed as Comirnaty and is fully approved for the prevention of COVID-19 in individuals aged ≥16 yrs. The companies also plan to …

Pfizer and BioNTech’s Comirnaty Receive the US FDA’s Approval to Prevent COVID-19 in Individuals Aged 16 Years and Older Read More »

Zydus’ ZyCoV-D Receives the DCGI’s EUA for the Treatment of COVID-19

Shots: The DCGI has granted a EUA for ZyCoV-D which is the world’s first plasmid DNA vaccine for COVID-19. The company is planning to seek approval for the two-dose regimen of the vaccine ZyCoV-D will become India’s first vaccine for adolescents aged 12-18 yrs. The vaccine is safe and well-tolerated ZyCoV-D is a three-dose, needle-free, …

Zydus’ ZyCoV-D Receives the DCGI’s EUA for the Treatment of COVID-19 Read More »

Pfizer’s Xeljanz (tofacitinib citrate) Receives EC’s Approval for the Treatment of Active Polyarticular JIA & Juvenile PsA

Shots: The approval is based on a P-III study which includes 2 phases i.e., run-in & withdrawal phase evaluating efficacy & safety of tofacitinib (5mg tablet or mg/mL oral solution, BID) in 398 patients aged ≥2yrs. with polyarticular JIA & juvenile PsA who have had an inadequate response to previous therapy with DMARDs for 44wks. …

Pfizer’s Xeljanz (tofacitinib citrate) Receives EC’s Approval for the Treatment of Active Polyarticular JIA & Juvenile PsA Read More »

BeiGene Reports NMPA’s Acceptance of sBLA for Tislelizumab (BGB-A317) to Treat Nasopharyngeal Cancer in China

Shots: The sBLA is based on P-III RATIONALE 309 trial evaluating the efficacy & safety of tislelizumab + CT vs PBO + CT in a ratio (1:1) in 263 Asian patients with recurrent or metastatic NPC The 1EPs of a trial is PFS as assessed by IRC in ITT population & 2EPs includes OS, ORR, …

BeiGene Reports NMPA’s Acceptance of sBLA for Tislelizumab (BGB-A317) to Treat Nasopharyngeal Cancer in China Read More »

BMS’ Opdivo(nivolumab) Receives the US FDA’s Approval for the Adjuvant Treatment of High-Risk Urothelial Carcinoma

Shots: The approval is based on the P-III CheckMate -274 trial evaluating Opdivo (240mg, IV, q2w, for ~1 yrs.) vs PBO in a ratio (1:1) in 709 patients with UC who are at high risk of recurrence after undergoing radical resection The results showed a 30% reduction in the risk of disease recurrence or death, …

BMS’ Opdivo(nivolumab) Receives the US FDA’s Approval for the Adjuvant Treatment of High-Risk Urothelial Carcinoma Read More »

Roche’s Ventana MMR RxDx Panel Test Receives the US FDA’s Approval as First CDx to Detect dMMR Solid Tumor

Shots: The FDA has approved the Ventana MMR RxDx Panel test as a CDx to identify patients with dMMR advanced solid tumors, who may be eligible for Jemperli monothx., based on the results of the MMR biomarker test Ventana MMR RxDx Panel provides access to clinicians with fully automated panel of MMR biomarkers tested by …

Roche’s Ventana MMR RxDx Panel Test Receives the US FDA’s Approval as First CDx to Detect dMMR Solid Tumor Read More »

Astellas and FibroGen’s Evrenzo (roxadustat) Receive EC’s Approval for Symptomatic Anemia Associated with Chronic Kidney Disease

Shots: The approval is based on the P-III program including 8 studies evaluating Evrenzo in 9600 patients with symptomatic anemia associated with CKD, regardless of dialysis status & prior ESA treatment. Following the EC’s approval, FibroGen to receive $120M milestones along with royalties based upon EU sales The results demonstrated that the therapy was effective …

Astellas and FibroGen’s Evrenzo (roxadustat) Receive EC’s Approval for Symptomatic Anemia Associated with Chronic Kidney Disease Read More »

Gilead Reports EMA’s Validation of MAA for Lenacapavir to Treat HIV-1 in People with Limited Therapy Options

Shots: The MAA is based on the ongoing P-II/III CAPELLA study evaluates the antiviral activity of lenacapavir (PO, SC, every 6mos.) in combination with other antiretroviral agents vs PBO in a ratio (2:1) in 36 patients with multi-drug resistant HIV-1 infection Results: viral load reduction from baseline of at least 0.5 log10 copies/mL for 14-day …

Gilead Reports EMA’s Validation of MAA for Lenacapavir to Treat HIV-1 in People with Limited Therapy Options Read More »

BMS’ Abecma (Idecabtagene Vicleucel) Receives EC’s Conditional Approval for Relapsed and Refractory Multiple Myeloma

Shots: The conditional approval is based on KarMMa study evaluating Abecma (single infusion with a target dose of 420 x 106 CAR T cells) in 128 patients with r/r MM prior treated with 3L therapies including an immunomodulatory agent, a proteasome inhibitor & an anti-CD38 Ab and who are refractory to their last treatment Results: …

BMS’ Abecma (Idecabtagene Vicleucel) Receives EC’s Conditional Approval for Relapsed and Refractory Multiple Myeloma Read More »

Does draft RCT guide set the scene for future trials?

A common set of standards for randomised clinical trials (RCTs) aims to help researchers develop the drugs and interventions of the future – no matter what they do or where they are based. The Good Clinical Trials Collaborative (GCTC) has published a draft guidance document, and is asking for the life sciences sector to make …

Does draft RCT guide set the scene for future trials? Read More »

Boehringer Ingelheim and Eli Lilly’s Jardiance (empagliflozin) Receive the US FDA’s Approval for the Treatment of Heart Failure with Reduced Ejection Fraction

Shots: The approval is based on the P-III EMPEROR-Reduced trial evaluating the effect of Jardiance (10 mg, qd) vs PBO in 3730 adults with HFrEF with/out T2D The results demonstrated a 25% reduction in the relative risk of the primary composite EPs of time to CV death or hospitalization for HF. The 2EPs demonstrated 30% …

Boehringer Ingelheim and Eli Lilly’s Jardiance (empagliflozin) Receive the US FDA’s Approval for the Treatment of Heart Failure with Reduced Ejection Fraction Read More »

BeiGene and EUSA Receive the NMPA’s Approval of Qarziba (dinutuximab beta) for High-Risk Neuroblastoma

Shots: The conditional approval is based on the SIOPEN trial evaluating dinutuximab beta vs dinutuximab beta with/out IL-2 in 514 patients aged ≥12 mos. with high-risk neuroblastoma who have received induction CT & partial response along with a history of R/R neuroblastoma with/out the residual disease. The trial was conducted by SIOPEN in collaboration with …

BeiGene and EUSA Receive the NMPA’s Approval of Qarziba (dinutuximab beta) for High-Risk Neuroblastoma Read More »

Agios Reports the US FDA’s Acceptance of NDA and Priority Review for Mitapivat to Treat Pyruvate Kinase Deficiency

Shots: The NDA & MAA submissions are based on ongoing 2 studies i.e., ACTIVATE & ACTIVATE-T evaluates safety, tolerability & efficacy of mitapivat vs PBO in patients with PKD Results: 40% of patients achieved a hemoglobin response, 33% reduction in transfusion burden @ 24wks. fixed dose period & safety profile was consistent with previously reported …

Agios Reports the US FDA’s Acceptance of NDA and Priority Review for Mitapivat to Treat Pyruvate Kinase Deficiency Read More »

GSK’s Jemperli (dostarlimab-gxly) Receives the US FDA’s Accelerated Approval for the Treatment of dMMR Recurrent or Advanced Solid Tumors

Shots: The approval is based on the results from cohort A1 & F of the ongoing P-I GARNET trial evaluates dostarlimab (500mg, IV, q3w for 4 doses) as monothx. in 209 patients with dMMR solid tumors, including EC & non-endometrial cancer. The therapy was discovered by AnaptysBio & licensed to Tesaro Results: In all dMMR …

GSK’s Jemperli (dostarlimab-gxly) Receives the US FDA’s Accelerated Approval for the Treatment of dMMR Recurrent or Advanced Solid Tumors Read More »

Global regulators seek permanent working group on AI

Rapid advances in the use of artificial intelligence (AI) in healthcare and medicines development are at risk of outstripping current regulatory frameworks, according to a new report. The International Coalition of Medicines Regulatory Authorities (ICMRA) has recommended that a new permanent working group be set up to keep tabs on the use of AI and …

Global regulators seek permanent working group on AI Read More »

BMS Reports EMA’s Validation of MAA for Opdivo (nivolumab) + Yervoy (ipilimumab) and Opdivo + CT to Treat Esophageal Squamous Cell Carcinoma

Shots: The applications are based on P-III CheckMate -648 trial evaluates Opdivo (3 mg/kg, q2w) + Yervoy (1 mg/kg, q6w up to 24mos.) or Opdivo (nivolumab) + CT vs CT alone in patients with unresectable advanced or metastatic ESCC Results: Both combinations showed OS benefit over CT at the pre-specified interim analysis in patients with …

BMS Reports EMA’s Validation of MAA for Opdivo (nivolumab) + Yervoy (ipilimumab) and Opdivo + CT to Treat Esophageal Squamous Cell Carcinoma Read More »

Novartis’ Cosentyx (secukinumab) Receives the NMPA’s Approval for the Treatment of Pediatric Psoriasis in China

Shots: The approval is based on two P-III international studies that evaluate cosentyx in pediatric patients aged 6 to <18 yrs. with mod. to sev. PsO who are candidates for systemic therapy or phototherapy The results showed that the therapy is safe and effective for children and adults with PsO. Additionally, therapy has supported the …

Novartis’ Cosentyx (secukinumab) Receives the NMPA’s Approval for the Treatment of Pediatric Psoriasis in China Read More »

Eli Lilly’s Lyumjev (insulin lispro-aabc injection) Receives the US FDA’s Expanded Label Approval for the Treatment of Type 1 and Type 2 Diabetes

Shots: The approval is based on P-III PRONTO-PUMP-2, a treat-to-target study that evaluates the efficacy & safety of Lyumjev (insulin lispro-aabc injection, 100 units/mL) vs Humalog (insulin lispro injection, 100 units/mL) in 432 adults in a ratio (1:1) with T1D for 16wks. The study met its 1EPs i.e., non-inferior A1C reduction from baseline @16wks. Lyumjev …

Eli Lilly’s Lyumjev (insulin lispro-aabc injection) Receives the US FDA’s Expanded Label Approval for the Treatment of Type 1 and Type 2 Diabetes Read More »

Merck Reports the Initiation of Rolling Submission to Health Canada for Molnupiravir to Treat COVID-19

Shots: The initiation of a rolling submission to Health Canada for molnupiravir (bid, for 5 days) to treat COVID-19 is based on the P-II interim results from the P-II/III MOVe-OUT clinical trials. The therapy is being developed in collaboration with Ridgeback The submission was accepted under the Minister of Health’s interim order and enables the …

Merck Reports the Initiation of Rolling Submission to Health Canada for Molnupiravir to Treat COVID-19 Read More »

Pfizer’s Ticovac Receives the US FDA’s Approval for the Prevention of Tick-Borne Encephalitis

Shots: The US FDA has approved Ticovac for active immunization to prevent TBE in patients aged ≥1 yrs. The US CDC’s will discuss recommendations regarding the safe and appropriate use of the vaccine In clinical trials, safety & immunogenicity of Ticovac were evaluated across two age groups (aged 1-15 yrs. and >16 yrs.). The studies …

Pfizer’s Ticovac Receives the US FDA’s Approval for the Prevention of Tick-Borne Encephalitis Read More »

The US FDA Rejects Sesen Bio’s Vicineum (oportuzumab monatox-qqrs) for the Treatment of BCG-Unresponsive NMIBC

Shots: The company has received the FDA’s CRL declining the approval of Vicineum’s BLA to treat BCG-unresponsive NMIBC The FDA has provided recommendations to conduct additional clinical/statistical data and analyses of roxadustat in addition to CMC issues related to a recent pre-approval inspection and product quality The company plans to meet with the FDA as …

The US FDA Rejects Sesen Bio’s Vicineum (oportuzumab monatox-qqrs) for the Treatment of BCG-Unresponsive NMIBC Read More »

Merck’s Welireg (belzutifan) Receives the US FDA’s Approval for the Treatment of Von Hippel-Lindau Disease Associated Tumors

Shots: The approval is based on open-label study 004 trial evaluates welireg (120 mg, qd) in patients with VHL-associated tumors including RCC, CNS hemangioblastomas, or pNET The results showed ORR (49%), m-DoR had not reached, 56% were still responding after 12mos., m-TTR (8mos.) in 61 patients with VHL-associated RCC, Additionally, 24 & 12 patients with …

Merck’s Welireg (belzutifan) Receives the US FDA’s Approval for the Treatment of Von Hippel-Lindau Disease Associated Tumors Read More »

Junshi and Coherus’s Toripalimab Receive the US FDA’s Breakthrough Therapy Designation as 1L Treatment of Nasopharyngeal Carcinoma

Shots: The designation is based on the P-III JUPITER-02 trial evaluating toripalimab + CT vs CT alone in patients with NPC. The result was presented at ASCO 2021 The results demonstrated an improvement in PFS. The trial also meets 2EPs of PFS assessed by the investigator and ORR as assessed by BIRC including higher DoR; …

Junshi and Coherus’s Toripalimab Receive the US FDA’s Breakthrough Therapy Designation as 1L Treatment of Nasopharyngeal Carcinoma Read More »

Celltrion’s Regdanvimab (CT-P59) Receives the ANVISA’s EUA for the Treatment of COVID-19 in Brazil

Shots: The EUA is based on P-III study evaluating regdanvimab vs PBO in adults with COVID-19 who do not require supplemental oxygen & are at high risk of progression to severe COVID-19 The results demonstrated a 72% reduction in risk of COVID-19 related hospitalization or death in high-risk patients & 70% for all patients, shortened …

Celltrion’s Regdanvimab (CT-P59) Receives the ANVISA’s EUA for the Treatment of COVID-19 in Brazil Read More »

Jazz’ Xywav Receives the US FDA’s Approval for the Treatment of Idiopathic Hypersomnia

Shots: The approval is based on the P-III withdrawal study that evaluates the efficacy and safety of Xywav (twice or once-nightly regimen, oral solution) vs PBO in adults with IH. The therapy is expected to be available in late 2021, following REMS implementation The results demonstrated the change in the 1EPs of ESS score & …

Jazz’ Xywav Receives the US FDA’s Approval for the Treatment of Idiopathic Hypersomnia Read More »

C4T’s CFT7455 Receives the US FDA’s Orphan Drug designation for the Treatment of Multiple Myeloma

Shots: The US FDA has granted ODD to CFT7455 for the treatment of MM. The FDA grants ODD to drugs & biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases, or conditions that affect fewer than 200,000 people in the US In Jun’21, the company has initiated a …

C4T’s CFT7455 Receives the US FDA’s Orphan Drug designation for the Treatment of Multiple Myeloma Read More »

Merck’s Keytruda (pembrolizumab) + Lenvima (lenvatinib) Receive the US FDA’s Approval as 1L Treatment of Advanced Renal Cell Carcinoma

Shots: The approval is based on P-III CLEAR /KEYNOTE-581 trial that evaluates Keytruda (200 mg, IV, q3w) + Lenvima (20 mg, PO, qd) or Lenvima (18mg) + everolimus (5mg, PO, qd) vs sunitinib (50 mg, q4w) in a ratio (1:1:1) in 1,069 patients with advanced RCC The results demonstrated an improvement in PFS with a …

Merck’s Keytruda (pembrolizumab) + Lenvima (lenvatinib) Receive the US FDA’s Approval as 1L Treatment of Advanced Renal Cell Carcinoma Read More »

The US FDA’s Rejects FibroGen’s Roxadustat for the Treatment of Anemia Due to CKD

Shots: The company has received an FDA’s CRL declining the approval of roxadustat’s NDA to treat anemia of CKD The FDA has requested to conduct an additional clinical study of roxadustat before resubmission The therapy is approved in China, Japan, Chile, and South Korea for the treatment of anemia of CKD in both NDD and …

The US FDA’s Rejects FibroGen’s Roxadustat for the Treatment of Anemia Due to CKD Read More »

Merck Reports the US FDA’s Acceptance of Keytruda’s sBLA for Review to Treat MSI-H/dMMR Advanced Endometrial Carcinoma

Shots: The application is based on ORR data from cohorts D & K of KEYNOTE-158 trial evaluating Keytruda as monothx. (200 mg, q3w) in 90 patients with MSI-H/dMMR advanced EC with disease progression following prior systemic therapy in any setting & are not candidates for curative surgery or radiation This indication is approved under accelerated …

Merck Reports the US FDA’s Acceptance of Keytruda’s sBLA for Review to Treat MSI-H/dMMR Advanced Endometrial Carcinoma Read More »

GW’s Epidyolex (cannabidiol) Receives MHRA’s Approval for the Treatment of Seizures Associated with Tuberous Sclerosis Complex in UK

Shots: The approval is based on P-III safety & efficacy study that evaluates Epidyolex (25 mg/kg/day) vs PBO in patients aged ≥2yrs. as an adjunctive treatment with seizures associated with TSC The study met its 1EPs i.e., reduction in seizure frequency (49% vs 27%). The 2EPs had supported the effects on 1EPs & safety profile …

GW’s Epidyolex (cannabidiol) Receives MHRA’s Approval for the Treatment of Seizures Associated with Tuberous Sclerosis Complex in UK Read More »

Merck Reports the US FDA’s Acceptance of sBLA and Priority Review for Keytruda (pembrolizumab) as an Adjuvant Treatment for RCC

Shots: The sBLA is based on P-III KEYNOTE-564 trial evaluating Keytruda monothx. (200mg, IV on day 1 of each 3wks. cycle for ~17 cycles) vs PBO in 994 patients with RCC, following nephrectomy and resection of metastatic lesions The results demonstrated an improvement in DFS and were presented at ASCO 2021 with an anticipated PDUFA …

Merck Reports the US FDA’s Acceptance of sBLA and Priority Review for Keytruda (pembrolizumab) as an Adjuvant Treatment for RCC Read More »

Calliditas Receives the US FDA’s Fast Track Designation for Setanaxib to Treat Primary Biliary Cholangitis

Shots: The US FDA has granted FTD to selinexor (NOX inhibitor) for the treatment of PBC. Additionally, the therapy has previously received ODD for PBC in the US & EU The P-II clinical trial of the therapy demonstrated anti-fibrotic activity as measured by Fibroscan and showed favorable tolerability profile along with the impact on fatigue …

Calliditas Receives the US FDA’s Fast Track Designation for Setanaxib to Treat Primary Biliary Cholangitis Read More »

AstraZeneca’s Forxiga (dapagliflozin) Receives EC’s Approval for the Treatment of Chronic Kidney Disease in Patients with and without T2D

Shots: The approval is based on a P-III DAPA-CKD trial that evaluates the efficacy of Forxiga (10mg, qd in addition to SoC) vs PBO in 4304 patients with CKD Stage 2-4 & elevated urinary albumin excretion with & without T2D The results showed 39% reduction in risk of worsening of renal function, ESKD, CV or …

AstraZeneca’s Forxiga (dapagliflozin) Receives EC’s Approval for the Treatment of Chronic Kidney Disease in Patients with and without T2D Read More »

Sanofi’s Nexviazyme (avalglucosidase alfa-ngpt) Receives the US FDA’s Approval for the Treatment of Late-Onset Pompe Disease

Shots: The approval is based on the P-III COMET trial that evaluates Nexviazyme (q2w, IV infusion) vs alglucosidase alfa in patients aged ≥1yrs. with LOPD for 49wks. Results: patients achieved noninferiority with 2.4 points greater improvement in FVC percent-predicted @49wks., were able to walk 32.2 m farther @49wks. on the 6MWT. The therapy showed improvements …

Sanofi’s Nexviazyme (avalglucosidase alfa-ngpt) Receives the US FDA’s Approval for the Treatment of Late-Onset Pompe Disease Read More »

Pfizer, Flynn in the firing line again over UK epilepsy drug pricing

The UK’s Competition and Markets Authority (CMA) has resurrected a complaint against Pfizer and Flynn Pharma, claiming the two companies charged the NHS excessive prices for a widely-used epilepsy therapy. In a provisional judgment, the CMA has concluded that Pfizer and Flynn abused a dominant position to overcharge the NHS for phenytoin sodium capsules, causing …

Pfizer, Flynn in the firing line again over UK epilepsy drug pricing Read More »

Merck’s Keytruda (pembrolizumab) Receives the US FDA’s Priority Review for the Adjuvant Treatment of Stage II Resected High-Risk Melanoma

Shots: The priority review is based on P-III KEYNOTE-716 trial assessing Keytruda (anti-PD-1 therapy) vs PBO in 954 patients aged ≥12yrs. with stage II resected high-risk melanoma. The anticipated PDUFA date for the therapy is Dec 04, 2021 The trial met its 1EPs of RFS with no new safety signals observed. Based on this data, …

Merck’s Keytruda (pembrolizumab) Receives the US FDA’s Priority Review for the Adjuvant Treatment of Stage II Resected High-Risk Melanoma Read More »

BeyondSpring Reports Results of Plinabulin + Docetaxel in P-III DUBLIN-3 Trial for 2L and 3L Treatment of NSCLC with EGFR Wild Type

Shots: The P-III DUBLIN-3 trial evaluates plinabulin + docetaxel vs docetaxel alone in 599 patients with 2L & 3L NSCLC EGFR wild type, with measurable lung lesion The trial met its 1EPs & 2EPs i.e improvement in OS, ORR, PFS, and OS rate @24 & 36mos. (22.1% vs 12.5% & 11.7% vs 5.3%); @48mos. OS …

BeyondSpring Reports Results of Plinabulin + Docetaxel in P-III DUBLIN-3 Trial for 2L and 3L Treatment of NSCLC with EGFR Wild Type Read More »

UCB’s Bimekizumab Receives NICE Recommendation for the Fast-Track Appraisal to Treat Severe Plaque Psoriasis

Shots: NICE has issued a final appraisal determination (FAD) which recommends UCB’s bimekizumab as a treatment option for adults with severe PsO The recommendation is based on four P-III studies i.e BE VIVID, BE RADIANT (P- IIIb), BE READY & BE SURE that demonstrated the superior efficacy and safety of bimekizumab in adults with PsO …

UCB’s Bimekizumab Receives NICE Recommendation for the Fast-Track Appraisal to Treat Severe Plaque Psoriasis Read More »

Roche Reports the US FDA’s Acceptance of BLA and Priority Review of Tecentriq (atezolizumab) as an Adjuvant Treatment for NSCLC

Shots: The application is based on the P-III IMpower010 study evaluating the efficacy & safety of Tecentriq vs BSC in 1005 patients in a ratio (1:1) with Stage IB-IIIA NSCLC, following surgical resection and up to 4 cycles of adjuvant cisplatin-based CT The results from an interim analysis showed the reduction in the risk of …

Roche Reports the US FDA’s Acceptance of BLA and Priority Review of Tecentriq (atezolizumab) as an Adjuvant Treatment for NSCLC Read More »

Janssen’s Uptravi (selexipag) Receives the US FDA’s Approval for IV Use in Adults with Pulmonary Arterial Hypertension

Shots: The approval is based on P-III UPTRAVI IV study evaluating the safety, tolerability, and PK of temporarily switching Uptravi (PO) to Uptravi (IV) in 20 patients with PAH The results showed that switching b/w Uptravi (PO) to Uptravi IV was well tolerated with no unexpected safety findings. Both formulations maintain the treatment effect for …

Janssen’s Uptravi (selexipag) Receives the US FDA’s Approval for IV Use in Adults with Pulmonary Arterial Hypertension Read More »

AstraZeneca’s Saphnelo (anifrolumab-fnia) Receives the US FDA’s Approval for the Treatment of Moderate to Severe Systemic Lupus Erythematosus

Shots: The approval is based on efficacy and safety data from the clinical development program, including two P- III TULIP trials and the P-II MUSE trial evaluating Saphnelo vs PBO in patients with SLE who are receiving standard therapy The result from the trials showed a reduction in overall disease activity across organ systems, including …

AstraZeneca’s Saphnelo (anifrolumab-fnia) Receives the US FDA’s Approval for the Treatment of Moderate to Severe Systemic Lupus Erythematosus Read More »

GSK’s Nucala (mepolizumab) Receives the US FDA’s Approval for the Treatment of Chronic Rhinosinusitis with Nasal Polyps

Shots: The approval is based on data from the SYNAPSE study evaluating mepolizumab vs PBO in ~400 patients with CRSwNP who had a history of prior surgery & need further surgery due to severe symptoms & increased size of polyps The results showed a 57% reduction in the size of nasal polyps & nasal obstruction …

GSK’s Nucala (mepolizumab) Receives the US FDA’s Approval for the Treatment of Chronic Rhinosinusitis with Nasal Polyps Read More »

UK fines Advanz £100m for raising thyroid drug price tenfold

The UK has levied another big fine for anticompetitive activity in the pharma market in a fortnight, with Advanz Pharma and former owners on the hook for more than £100 million ($140 million) after increasing the price of a thyroid disease drug by 1,110% over an eight-year period. The Competition and Markets Authority (CMA) said …

UK fines Advanz £100m for raising thyroid drug price tenfold Read More »

Takeda’s TAK-994 Receives Breakthrough Therapy Designation for the Treatment of Narcolepsy Type 1

Shots: The BTD is based on early and preliminary clinical data involves assessing TAK-994 for the treatment of EDS that may demonstrated substantially improved objective and subjective measurements of daytime wakefulness in NT1 patients TAK-994 is being evaluated in an ongoing P-II TAK-994-1501 study while the completed data from study will be presented at a …

Takeda’s TAK-994 Receives Breakthrough Therapy Designation for the Treatment of Narcolepsy Type 1 Read More »

Roche Reports the US FDA’s Acceptance of BLA for Faricimab to Treat Diabetic Macular Edema and Neovascular Age-Related Macular Degeneration

Shots: The BLA submission is based on four P-III studies i.e TENAYA, LUCERNE, YOSEMITE & RHINE evaluating the efficacy & safety of faricimab vs aflibercept in patients with nAMD & DME. The LTE studies for faricimab are underway The studies demonstrated non-inferior visual acuity gains and greater reductions in CST, ~50% of patients can extend …

Roche Reports the US FDA’s Acceptance of BLA for Faricimab to Treat Diabetic Macular Edema and Neovascular Age-Related Macular Degeneration Read More »

Sanofi’s Avalglucosidase alfa Receives CHMP’s Positive Opinion for Patients with Pompe Disease

Shots: The CHMP’s positive opinion is based on P-III COMET study assessing avalglucosidase alfa that demonstrated improvements in respiratory function & movement endurance measures in people with late-onset Pompe disease The CHMP concluded that avalglucosidase alfa does not qualify as a New Active Substance (NAS). Sanofi will be requesting a re-examination of the CHMP’s opinion …

Sanofi’s Avalglucosidase alfa Receives CHMP’s Positive Opinion for Patients with Pompe Disease Read More »

GSK’s Shingrix Receives the US FDA’s Approval to Prevent Shingles in Immunocompromised Adults

Shots: The approval is based on studies evaluating the safety & efficacy of Shingrix in adults aged ≥18yrs. who had undergone auHSCT and those undergoing treatment for hematological malignancies (post-hoc analysis) The safety & immunogenicity data were generated in adults who were or anticipated to be, immunodeficient/immunosuppressed due to known therapy including patients with HIV, …

GSK’s Shingrix Receives the US FDA’s Approval to Prevent Shingles in Immunocompromised Adults Read More »

BeiGene’s Brukinsa (Zanubrutinib) Receives Health Canada’s Approval for Mantle Cell Lymphoma

Shots: The approval is based on efficacy results from two studies. The P-II BGB-3111-206 study evaluates zanubrutinib in patients with R/R MCL and showed ORR (84%), CR (69%), PR (15%), mDoR (19.5mos.) @median follow-up time of 18.5mos. The P-I/II BGB-3111-AU-003 study showed ORR (84%) including CR (25%) & PRs (59%), m-DoR (18.5mos.) @ median follow-up …

BeiGene’s Brukinsa (Zanubrutinib) Receives Health Canada’s Approval for Mantle Cell Lymphoma Read More »

The US FDA Declines to Approve Incyte’s Retifanlimab to Treat Patients with Squamous Cell Carcinoma of the Anal Canal

Shots: The US FDA has issued a CRL to the BLA of retifanlimab. The BLA submission is based on P-II POD1UM-202 trial assessing retifanlimab in prior treated patients with LA or m-SCAC who have progressed on or were ineligible for or intolerant of Pt. based CT The FDA stated that an additional data is needed …

The US FDA Declines to Approve Incyte’s Retifanlimab to Treat Patients with Squamous Cell Carcinoma of the Anal Canal Read More »

Alexion’s Ultomiris (ravulizumab) Receives CHMP’s Positive Opinion Recommending its Approval for Children and Adolescents with PNH

Shots: The CHMP’s positive opinion is based on a P-III study assessing Ultomiris and showed effectiveness in completing C5 complement inhibition @26wks. for children & adolescents aged ≤18yrs. with PNH The study demonstrated that there is no reported treatment-related sAEs, and no patients discontinued treatment during the primary evaluation period or experienced breakthrough hemolysis The …

Alexion’s Ultomiris (ravulizumab) Receives CHMP’s Positive Opinion Recommending its Approval for Children and Adolescents with PNH Read More »

AstraZeneca’s Bydureon BCise (exenatide extended-release) Receives the US FDA’s Approval for the Treatment of Type2 Diabetes

Shots: The approval is based on results of the P-III BCB114 trial evaluating Bydureon BCise (qw, injectable suspension) vs PBO in 82 patients aged b/w 10 – 18 yrs. with T2D as an adjuvant to diet and exercise alone or in combination with a stable dose of oral antidiabetic agents and insulin for 24 wks. …

AstraZeneca’s Bydureon BCise (exenatide extended-release) Receives the US FDA’s Approval for the Treatment of Type2 Diabetes Read More »

Merck and Eisai’s Keytruda (pembrolizumab) + Lenvima (lenvatinib) Receive the US FDA’s Approval for the Treatment of Advanced Endometrial Carcinoma

Shots: The approval is based on P-III KEYNOTE-775/Study 309 trial evaluating Merck’s Keytruda (200mg, IV, q3w) + Eisai’s Lenvima (20mg, PO, qd) vs CT (doxorubicin, 60 mg/m2, IV, q3wks.) or paclitaxel (80 mg/m2, IV, 28-day cycle) in 827 patients with advanced EC that were not MSI-H or dMMR, prior treated with Pt-based regimen in any …

Merck and Eisai’s Keytruda (pembrolizumab) + Lenvima (lenvatinib) Receive the US FDA’s Approval for the Treatment of Advanced Endometrial Carcinoma Read More »

Bluebird Bio’s Skysona (elivaldogene autotemcel) Receives EC’s Approval for the Treatment of Cerebral Adrenoleukodystrophy

Shots: The approval is based on P-II/III ALD-102 study evaluating Skysona in patients with an ABCD1 gene mutation aged <18 yrs. with CALD without matched sibling donors. The P-III ALD-104 study is ongoing Results: 90% of patients met the MFD-free survival 1EPs @24mos., 26 out of 28 patients maintained an NFS ≤1 @24mos. All patients …

Bluebird Bio’s Skysona (elivaldogene autotemcel) Receives EC’s Approval for the Treatment of Cerebral Adrenoleukodystrophy Read More »

Mabpharm’s Biobetter (CMAB008) for Infliximab Receives NMPA’s Approval in China

Shots: NMPA has granted approval to market infliximab biobetter in China. Sorrento holds exclusive commercial rights to Infliximab biobetter Ab outside of China and plans to file a BLA for the product in the US and EU in 2021 Biobetters are drugs that have similar structure and properties to an innovator product but are enhanced …

Mabpharm’s Biobetter (CMAB008) for Infliximab Receives NMPA’s Approval in China Read More »

Bayer and Merck’s Verquvo (vericiguat) Receives the EC’s Approval for the Treatment of Chronic Heart Failure

Shots: The EC has approved Verquvo (2.5/5/10 mg) in the EU for the treatment of symptomatic CHF in adult patients with reduced ejection fraction who are stabilized after a recent decompensation event. The therapy is being jointly developed by both companies Merck has the commercial rights to Verquvo in the US and Bayer has the …

Bayer and Merck’s Verquvo (vericiguat) Receives the EC’s Approval for the Treatment of Chronic Heart Failure Read More »

Genentech’s Venclexta (venetoclax) + Azacitidine Receives Breakthrough Therapy Designation for the Treatment of Myelodysplastic Syndromes

Shots: The designation is based on interim results from the P-Ib M15-531 study evaluating Venclexta + azacitidine in naïve patients with previously untreated higher-risk MDS The primary objectives of the study are to assess the safety, Pk and determine the RP2D and dosing schedule of Venclexta in combination with azacitidin Venclexta is a novel inhibitor …

Genentech’s Venclexta (venetoclax) + Azacitidine Receives Breakthrough Therapy Designation for the Treatment of Myelodysplastic Syndromes Read More »

Albireo’s Bylvay (odevixibat) Receives the US FDA’s Approval for the Treatment of Progressive Familial Intrahepatic Cholestasis

Shots: The approval is based on P-III PEDFIC 1 & 2 studies evaluating Bylvay (qd) vs PBO in patients with pruritus in PFIC. The PEDFIC 1 study met its 1EPs i.e the therapy showed a low incidence of diarrhea or frequent bowel movements (9.5% vs 5.0%) The PEDFIC 2 study showed sustained reductions in serum …

Albireo’s Bylvay (odevixibat) Receives the US FDA’s Approval for the Treatment of Progressive Familial Intrahepatic Cholestasis Read More »

Myovant’s Ryeqo (relugolix, estradiol, and norethindrone acetate) Receives EC’s Approval for the Treatment of Women with Uterine Fibroids

Shots: The approval is based on safety & efficacy data from the P-III LIBERTY program that consists of 2 studies i.e LIBERTY 1 & 2 evaluating Ryeqo in adult women with UF with no limitation for a duration of use. The EC’s decision is valid in all 27 member states of the EU, Iceland, Norway …

Myovant’s Ryeqo (relugolix, estradiol, and norethindrone acetate) Receives EC’s Approval for the Treatment of Women with Uterine Fibroids Read More »

FDA gives speedy review to Bayer’s Parkinson’s stem cell therapy

Bayer subsidiary BlueRock Therapeutics has been granted a fast-track review by the FDA for DA01, its stem cell-based therapy for Parkinson’s disease which is currently in early-stage clinical testing. The FDA designation allows for benefits such as more frequent meetings and communication with the regulator during clinical development, and a truncated six-month review time. Those …

FDA gives speedy review to Bayer’s Parkinson’s stem cell therapy Read More »

Kadmon’s Rezurock (belumosudil) Receives the US FDA’s Approval for the Treatment of Chronic Graft Versus Host Disease

Shots: The approval is based on KD025-213 study evaluating Rezurock (200 mg, qd) aged >12 yrs. in 65 patients with cGVHD who had received 2 to 5 prior lines of systemic therapy. The anticipated PFUFA date is Aug 30, 2021 Results: ORR (75%) through Cycle 7, day 1 treatment with CR (6%) & PR (69%); …

Kadmon’s Rezurock (belumosudil) Receives the US FDA’s Approval for the Treatment of Chronic Graft Versus Host Disease Read More »

Regeneron’s Ronapreve Antibody Cocktail (casirivimab and imdevimab) Receives MHLW’s Approval for the Treatment of Mild to Moderate COVID-19

Shots: The approval is based on the results from a P-III trial evaluating Ronapreve (casirivimab and imdevimab) in high-risk non-hospitalized patients with COVID-19 The results showed a reduction in the risk of hospitalization or death by 70% in addition to the results from a P-I trial that demonstrated the safety, tolerability & PK in Japanese …

Regeneron’s Ronapreve Antibody Cocktail (casirivimab and imdevimab) Receives MHLW’s Approval for the Treatment of Mild to Moderate COVID-19 Read More »

AstraZeneca’s Imfinzi (durvalumab) Receives NMPA’s Approval as a 1L Treatment of Extensive-Stage Small Cell Lung Cancer

Shots: The approval is based on P-III CASPIAN trial assessing Imfinzi + SoC (etoposide & carboplatin/ cisplatin CT) or Imfinzi + CT + tremelimumab vs CT alone in 805 patients with ES-SCLC in 200 centers across 23 countries Results: the study met its 1EPs of OS in Jun’19, demonstrating a 27% reduction in risk of …

AstraZeneca’s Imfinzi (durvalumab) Receives NMPA’s Approval as a 1L Treatment of Extensive-Stage Small Cell Lung Cancer Read More »

Sanofi’s Fexinidazole Receives the US FDA’s Approval as the First Oral Treatment for Sleeping Sickness

Shots: The US FDA has approved fexinidazole (PO, qd, 10days treatment) for both stages of Trypanosoma brucei gambiense form of sleeping sickness in patients aged >6yrs. & weighing at least 20 kg. The therapy has developed under the collaboration with DNDi, DRC, CAR & Sanofi Sanofi & partners are committed to access fexinidazole in all …

Sanofi’s Fexinidazole Receives the US FDA’s Approval as the First Oral Treatment for Sleeping Sickness Read More »

Merck’s Vaxneuvance (pneumococcal 15-valent conjugate vaccine) Receives the US FDA’s Approval for the Prevention of Invasive Pneumococcal Disease Caused by 15 Serotypes

Shots: The approval is based on seven P-II & III clinical studies involves assessing the safety, tolerability & immunogenicity of Vaxneuvance vs PCV13 in 7,438 adults aged >18yrs. with IPD caused by 15 serotypes Results: Vaxneuvance showed a non-inferior immune response to PCV13 for 13 shared serotypes as assessed by OPA GMTs & superior immune …

Merck’s Vaxneuvance (pneumococcal 15-valent conjugate vaccine) Receives the US FDA’s Approval for the Prevention of Invasive Pneumococcal Disease Caused by 15 Serotypes Read More »

UK fines drugmakers record £260m for price gouging NHS

The UK’s competition authority has issued its largest ever fine of more than £260 million ($360 million) to several pharma companies accused of colluding to hike the price of medicines delivered to the NHS. The Competition and Markets Authority (CMA) took the action against Auden Mckenzie and Actavis UK – now known as Accord-UK – …

UK fines drugmakers record £260m for price gouging NHS Read More »

FDA staffers defend Aduhelm approval in JAMA editorial

Three FDA figures at the centre of the fallout over the approval Biogen and Eisai’s Alzheimer’s disease therapy Aduhelm have defended their actions, arguing that to do otherwise would have left millions of patients in limbo for years. The trio say that the decision to approve Aduhelm (aducanumab) was extremely challenging as “evidence was strongly …

FDA staffers defend Aduhelm approval in JAMA editorial Read More »

Final hurdle falls as UK backs AZ’s $39bn takeover of Alexion

The UK’s competition regulator has approved AstraZeneca’s $39 billion acquisition of US-based drugmaker Alexion, without opting for a broader inquiry into the deal. The nod from the Competition and Markets Authority (CMA) after an initial assessment means that it concluded no antitrust risks were associated with the deal. It was the final needed from a …

Final hurdle falls as UK backs AZ’s $39bn takeover of Alexion Read More »

Janssen’s Darzalex Faspro (daratumumab and hyaluronidase-fihj) + Pomalidomide and Dexamethasone Receive the US FDA’s Approval to Treat Patients with Multiple Myeloma

Shots: The approval is based on P-III APOLLO study evaluating Darzalex Faspro (SC) + pomalidomide & dexamethasone (Pd) vs Pd alone in 304 patients with RRMM who have received at least one prior treatment regimen including lenalidomide & a proteasome inhibitor The study met its 1EPs of PFS & showed 37% reduction in risk of …

Janssen’s Darzalex Faspro (daratumumab and hyaluronidase-fihj) + Pomalidomide and Dexamethasone Receive the US FDA’s Approval to Treat Patients with Multiple Myeloma Read More »

Astellas and Seagen’s Padcev (enfortumab vedotin-ejfv) Receive the US FDA’s Full Approval and Expands Indication for Patients with Locally Advanced or Metastatic Urothelial Cancer

Shots: The approval is based on P-III EV-301 trial evaluating Padcev vs CT in 608 patients with LA or mUC who were previously treated with Pt.-based CT and a PD-1/L1 inhibitor In a pre-specified interim analysis, patients who received Padcev have m-OS (12.9 vs 9.0 mos.) i.e. 3.9mos. longer than those who received CT Padcev …

Astellas and Seagen’s Padcev (enfortumab vedotin-ejfv) Receive the US FDA’s Full Approval and Expands Indication for Patients with Locally Advanced or Metastatic Urothelial Cancer Read More »

Bayer’s Kerendia (finerenone) Receives the US FDA’s Approval for the Treatment of Chronic Kidney Disease Associated with Type 2 Diabetes

Shots: The approval is based on a P-III FIDELIO-DKD study evaluating finerenone (10/20mg) + SoC vs PBO + SoC in ~ 5700 patients with CKD & T2D. Patients were followed for a median of 2.6yrs. Results: reduction in the incidence of the primary composite EPs of a sustained decline in eGFR of ≥40%, reduction in …

Bayer’s Kerendia (finerenone) Receives the US FDA’s Approval for the Treatment of Chronic Kidney Disease Associated with Type 2 Diabetes Read More »

Lysogene Receives the US FDA’s Fast Track Designation for LYS-GM101 Gene Therapy to Treat GM1 Gangliosidosis

Shots: The FDA has granted FTD to LYS-GM101 for the Treatment of GM1 Gangliosidosis. The company has recently initiated an adaptative-design clinical trial evaluating the efficacy & safety of LYS-GM101 in 16 patients with GM1 gangliosidosis across the US & EU The P-II/III clinical trial for MPS IIIA is ongoing, following collaboration with Sarepta whereas …

Lysogene Receives the US FDA’s Fast Track Designation for LYS-GM101 Gene Therapy to Treat GM1 Gangliosidosis Read More »

Jazz’s Vyxeos (daunorubicin and cytarabine liposome) for Injection Receives Health Canada Approval for the Treatment of High-risk Acute Myeloid Leukemia

Shots: The approval is based on the results from a P-III study that involves assessing Vyxeos (IV infusion) vs a current conventional treatment group in 309 adult patients aged 60-75 yrs. with newly diagnosed t-AML or AML-MRC The study met its 1EPs i.e the therapy demonstrates the improvement in OS; m-OS (9.6 vs 6.0 mos.) …

Jazz’s Vyxeos (daunorubicin and cytarabine liposome) for Injection Receives Health Canada Approval for the Treatment of High-risk Acute Myeloid Leukemia Read More »

BeiGene’s Kyprolis (carfilzomib) Receives NMPA’s Conditional Approval for the Treatment of Relapsed or Refractory Multiple Myeloma

Shots: The approval is based on P-III trial evaluating efficacy & safety of Kyprolis + dexamethasone in 123 patients with RRMM who have received at least 2 prior therapies, including a proteasome inhibitor & an immunomodulatory agent Results: ORR (35.8%); m-PFS (5.6mos.) as assessed by IRC. Additionally, results showed that the efficacy & safety in …

BeiGene’s Kyprolis (carfilzomib) Receives NMPA’s Conditional Approval for the Treatment of Relapsed or Refractory Multiple Myeloma Read More »

AstraZeneca and Amgen Report the US FDA’s Acceptance of BLA and Priority Review of Tezepelumab for the Treatment of Asthma

Shots: The BLA is based on PATHFINDER clinical program, including P-III NAVIGATOR study evaluating Tezepelumab + SOC vs PBO + SOC in 1,061 adults (18–80yrs.) & adolescents (12–17yrs.) with severe & uncontrolled asthma who receive treatment with medium/high dose ICS + at least 1 additional controller medication with/out OCS The results demonstrated superiority in 1EP …

AstraZeneca and Amgen Report the US FDA’s Acceptance of BLA and Priority Review of Tezepelumab for the Treatment of Asthma Read More »

BeiGene Reports NMPA’s Acceptance of sBLA for Tislelizumab to Treat Esophageal Squamous Cell Carcinoma in China

Shots: The sBLA is based on P-III RATIONALE 302 trial evaluating efficacy & safety of tislelizumab vs CT as a 2L treatment in 512 patients in a ratio (1:1) with locally advanced or metastatic ESCC across 11 countries The 1EPs of a trial is OS in ITT populations & 2EPs is OS in patients with …

BeiGene Reports NMPA’s Acceptance of sBLA for Tislelizumab to Treat Esophageal Squamous Cell Carcinoma in China Read More »

Y-mAbs Reports BLA Submission to the NMPA for Danyelza (naxitamab-gqgk) to Treat Relapsed/Refractory High-Risk Neuroblastoma

Shots: SciClone has submitted the BLA for Danyelza to treat pediatric patients aged >1 yr. and adult patients with r/r high-risk neuroblastoma to NMPA in China If approved, the therapy will be available in the China imminently. The therapy was approved under accelerated approval based on ORR and DoR while the continued approval for the …

Y-mAbs Reports BLA Submission to the NMPA for Danyelza (naxitamab-gqgk) to Treat Relapsed/Refractory High-Risk Neuroblastoma Read More »

Merck’s Keytruda (pembrolizumab) Receives the US FDA’s Approval for Expanded Indication in Patients with Locally Advanced Cutaneous Squamous Cell Carcinoma

Shots: The approval is based on data from the second interim analysis of the P-II KEYNOTE-629 trial assessing Keytruda (200mg, IV, q3w) in 159 patients with recurrent, metastatic, or LA cSCC that is not curable by surgery or radiation Results: ORR (50%); CR (17%); PR (33%), 81% had DOR of 6mos. or longer, and 37% …

Merck’s Keytruda (pembrolizumab) Receives the US FDA’s Approval for Expanded Indication in Patients with Locally Advanced Cutaneous Squamous Cell Carcinoma Read More »

Immunic’s IMU-838 Receives the US FDA’s IND Approval to Initiate P-III ENSURE & P-II CALLIPER Studies for RRMS & PMS

Shots: The P-III ENSURE (1 &2) program evaluates IMU-838 (30mg, qd) vs PBO in ~ 1,050 patients with active RMS. The 1EPs of both trials is time to 1st relapse @72wks. & 2EPs include a volume of T2-lesions, time to confirm disability progression, changes in cognition & measure brain volume The P-II CALLIPER trial assess …

Immunic’s IMU-838 Receives the US FDA’s IND Approval to Initiate P-III ENSURE & P-II CALLIPER Studies for RRMS & PMS Read More »

Lupin’s Solosec (secnidazole) Receives the US FDA’s Approval for the Treatment of Trichomoniasis

Shots: The approval is based on a P-III clinical trial that involves assessing Solosec (2g, PO, granules) vs PBO in 64 patients with trichomoniasis The P-III trial demonstrated 92.2% cure rate in the modified ITT population and 94.9% in the per-protocol population, therapy was well-tolerated with no serious AEs were identified in the trial Additionally, …

Lupin’s Solosec (secnidazole) Receives the US FDA’s Approval for the Treatment of Trichomoniasis Read More »

Hutchmed Reports the US FDA’s Acceptance of Surufatinib’s NDA for Advanced Neuroendocrine Tumors

Shots: The NDA is based on two P-III studies of surufatinib in patients with pancreatic and extra-pancreatic NET in China. The anticipated PDUFA date is April 30, 2022 The same data from the studies will be used to file MAA to the EMA imminently, based on scientific advice from the EMA’s Committee for Medicinal Products …

Hutchmed Reports the US FDA’s Acceptance of Surufatinib’s NDA for Advanced Neuroendocrine Tumors Read More »

Zydus Seeks EUA for ZyCoV-D Against COVID-19 in India

Shots: The company has applied for emergency use authorization to the DCGI for ZyCoV-D, which is the world’s first plasmid DNA vaccine for COVID-19 Zydus conducted a clinical trial for its ZyCoV-D in the adolescent population aged 12-18yrs. in India over 50 centers. The vaccine is safe and well-tolerated and showed 66% efficacy for symptomatic …

Zydus Seeks EUA for ZyCoV-D Against COVID-19 in India Read More »

The US FDA Approves FoundationOne CDx for Alunbrig (brigatinib) in Patients with ALK+ NSCLC

Shots: The US FDA has approved FoundationOne CDx to be used as a CDx for Alunbrig (brigatinib) which is approved for the treatment of adult patients with ALK+ mNSCLC as detected by an FDA-approved test In Sept’20, Foundation Medicine and Takeda collaborated to develop CDx for therapies in its late-stage lung portfolio FoundationOne CDx is …

The US FDA Approves FoundationOne CDx for Alunbrig (brigatinib) in Patients with ALK+ NSCLC Read More »

Jazz’s Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn) Receives the US FDA’s Approval for Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma

Shots: The approval is based on clinical data from an ongoing P-II/III JZP458-201 trial evaluating Rylaze in pediatric and adult patients aged ≥1mos. with ALL or LBL who are allergic to the E. coli-derived asparaginase. Rylaze has approved under the RTOR program Additionally, recent data from a Children’s Oncology Group retrospective analysis in ~8,000 patients …

Jazz’s Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn) Receives the US FDA’s Approval for Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma Read More »

Abbott’s XIENCE Stent Receives the US FDA’s Approval for Shortest Blood Thinner Course in Patients with High Bleeding Risk

Shots: Abbott has received approval in the US & and CE mark for XIENCE stents, the shortest approved DAPT labeling as short as 28 days in patients with high bleeding risk. XIENCE Skypoint stent also received FDA approval in the US and CE Mark in the EU Additionally, patients with stents are typically on DAPT …

Abbott’s XIENCE Stent Receives the US FDA’s Approval for Shortest Blood Thinner Course in Patients with High Bleeding Risk Read More »

LEXEO Therapeutics’ LX2006 Receives the US FDA’s Rare Pediatric Disease Designation and Orphan Drug Designation to Treat Friedreich Ataxia

Shots: The US FDA has granted RPD and ODD to LX2006 for the treatment of FA. The company plans to start a P-I/II clinical trial of LX2006 in patients with cardiomyopathy associated with FA in 2021 Additionally, if BLA for LX2006 is approved, a company may be eligible to receive PRV that may be sold …

LEXEO Therapeutics’ LX2006 Receives the US FDA’s Rare Pediatric Disease Designation and Orphan Drug Designation to Treat Friedreich Ataxia Read More »

Formycon and Bioeq Report MAA Submission to EMA for FYB201 (biosimilar, ranibizumab)

Shots: Formycon and Bioeq have submitted MAA for FYB201 (biosimilar referencing Lucentis) to EMA Teva will responsible for the commercialization of FYB201 in the EU which has acquired the distribution rights under its collaboration with Bioeq. Currently, Formycon has four biosimilars in development Formycon focuses to treat ophthalmology, immunology, and other key chronic diseases covering …

Formycon and Bioeq Report MAA Submission to EMA for FYB201 (biosimilar, ranibizumab) Read More »

Blueprint Medicines’ Ayvakit (avapritinib) Receives FDA’s Approval to Treat Patients with Advanced Systemic Mastocytosis

Shots: The full FDA approval is based on data from 2 clinical trials i.e., P-I EXPLORER & P-II PATHFINDER trial evaluating the efficacy of Ayvakit (200 mg, PO, qd) in 53 adults with advanced SM including ASM, SM-AHN & MCL Results: @median follow-up of 11.6 mos., ORR (57%), CR/CRh (28%), PR (28 %) & clinical …

Blueprint Medicines’ Ayvakit (avapritinib) Receives FDA’s Approval to Treat Patients with Advanced Systemic Mastocytosis Read More »

MannKind and United Therapeutics Reports the US FDA’s Acceptance of NDA for Priority Review of Tyvaso DPI to Treat PAH and PH-ILD

Shots: The NDA is based on data from the BREEZE study evaluating Tyvaso DPI in 51 patients with PAH transitioning from Tyvaso inhalation solution which demonstrates the safety & tolerability of Tyvaso DPI Additionally, a separate PK study in 36 healthy volunteers demonstrates comparable treprostinil exposure b/w Tyvaso DPI & Tyvaso Inhalation Solution while FDA …

MannKind and United Therapeutics Reports the US FDA’s Acceptance of NDA for Priority Review of Tyvaso DPI to Treat PAH and PH-ILD Read More »

BeiGene’s Tislelizumab in Combination with Asieris’ APL-1202 Receive the US FDA’s IND Approval for MIBC

Shots: The US FDA has approved the IND application of oral Asieris’ APL-1202 + BeiGene’s tislelizumab as neoadjuvant therapy in patients with MIBC The P-I/II study will evaluate the safety of the dual regimen in MIBC patients, determine the RP2D and assess the efficacy of combo as neoadjuvant therapy for MIBC. Asieris will accelerate the …

BeiGene’s Tislelizumab in Combination with Asieris’ APL-1202 Receive the US FDA’s IND Approval for MIBC Read More »

Gilead’s Epclusa (Sofosbuvir/Velpatasvir) Receives the US FDA’s Approval for Children with Chronic Hepatitis C Infection

Shots: The US FDA has approved the NDA of an oral pellet formulation of Epclusa (sofosbuvir 200 mg/velpatasvir 50 mg and sofosbuvir 150 mg/velpatasvir 37.5 mg) for children aged 3 yrs. who cannot swallow tablets The approval is based on a P-II trial evaluating Epclusa in 41 children with HCV for 12 wks. The study …

Gilead’s Epclusa (Sofosbuvir/Velpatasvir) Receives the US FDA’s Approval for Children with Chronic Hepatitis C Infection Read More »

Vertex’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) Receives the US FDA’s Approval in Children Aged 6 to 11 with Certain Mutations in Cystic Fibrosis

Shots: The approval is based on a P-III study evaluating the safety, PK & efficacy of Trikafta in 66 children aged 6-11yrs. old with CF with either two copies of F508del mutations or one copy of F508del mutation & one minimal function mutation for 24wks. The company has filed the application in EMA & MHRA …

Vertex’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) Receives the US FDA’s Approval in Children Aged 6 to 11 with Certain Mutations in Cystic Fibrosis Read More »

Double resignation over Aduhelm approval depletes FDA’s CNS adcomm

Two members of an FDA advisory committee have now resigned in protest over Monday’s approval of Biogen’s Alzheimer’s disease drug Aduhelm despite an overwhelming vote against doing so. Washington University neurologist Joel Perlmutter was the first to announce he was stepping down from the Peripheral and Central Nervous System Drugs Advisory Committee on the day …

Double resignation over Aduhelm approval depletes FDA’s CNS adcomm Read More »

Pfizer’s Prevnar 20 (Pneumococcal 20-Valent Conjugate Vaccine) Receives the US FDA’s Approval for the Prevention of Invasive Disease and Pneumonia

Shots: The approval is based in the clinical program including P-I & II trials, and three P-III trials evaluating the safety & immunogenicity of Prevnar 20 in adults aged ≥18yrs. with invasive disease and pneumonia caused by the 20 S. pneumoniae serotypes This is the first approval of a vaccine that protects against 20 serotypes …

Pfizer’s Prevnar 20 (Pneumococcal 20-Valent Conjugate Vaccine) Receives the US FDA’s Approval for the Prevention of Invasive Disease and Pneumonia Read More »

AbbVie’s Rinvoq (upadacitinib) Receives the Health Canada’s Approval for the Treatment of Psoriatic Arthritis

Shots: The approval is based on data from two P-III studies i.e SELECT-PsA 1 & 2 evaluating the safety & efficacy of Rinvoq vs PBO in 2,000+ patients with active PsA who have had an inadequate response to methotrexate or other DMARDs The studies met their 1EPs of ACR20 response @12wks., Rinvoq (15mg) showed non-inferiority …

AbbVie’s Rinvoq (upadacitinib) Receives the Health Canada’s Approval for the Treatment of Psoriatic Arthritis Read More »

BeiGene Reports NMPA’s Acceptance of sBLA for Tislelizumab to Treat MSI-H or dMMR Solid Tumors

Shots: The sBLA submission is based on results from a P-II study evaluating the efficacy & safety of tislelizumab (200 mg, IV, q3w) as monothx in 80 patients with previously treated LA, unresectable or metastatic MSI-H or dMMR solid tumors The results demonstrated that the therapy treatment was consistent & durable across tumor types and …

BeiGene Reports NMPA’s Acceptance of sBLA for Tislelizumab to Treat MSI-H or dMMR Solid Tumors Read More »

Roche’s SARS-CoV-2 Antigen Test Receives CE mark For Self Testing of COVID-19 at Home

Shots: Roche received a CE mark for its SARS-CoV-2 Ag self-test nasal at home setting for COVID 19. The test will be available in countries accepting the CE mark The early version of the test is available in the EU under special approval pathways since Feb, 2021. The test can be performed by individuals using …

Roche’s SARS-CoV-2 Antigen Test Receives CE mark For Self Testing of COVID-19 at Home Read More »

Biogen and Eisai’s Aduhelm (aducanumab-avwa) Receive the US FDA’s Accelerated Approval for Alzheimer Disease

Shots: The approval is based on the two P-III clinical trials i.e EMERGE and ENGAGE along with P-Ib PRIME study involves assessing the efficacy of Aduhelm vs PBO in patients with early stages of AD with confirmed presence of amyloid pathology Results: the therapy consistently showed a dose and time-dependent effect on the reduction of …

Biogen and Eisai’s Aduhelm (aducanumab-avwa) Receive the US FDA’s Accelerated Approval for Alzheimer Disease Read More »

Novo Nordisk’s Wegovy (semaglutide, 2.4mg) Receives the US FDA’s Approval for Weight Management in Adults with Obesity

Shots: The approval is based on the results from the P-IIIa STEP clinical trial program evaluating Wegovy (2.4mg injection, qw) in ~4,500 adults with obesity or overweight with at least one weight-related comorbidity Results: the therapy showed 17-18% average weight loss in people without T2D @68wks. and has a safe and well-tolerated profile across the …

Novo Nordisk’s Wegovy (semaglutide, 2.4mg) Receives the US FDA’s Approval for Weight Management in Adults with Obesity Read More »

ProMetic’ Ryplazim (plasminogen) Receives the US FDA’s Approval as First Treatment for Patients with Plasminogen Deficiency

Shots: The approval is based on the study assessing the effectiveness and safety of Ryplazim (every two to four days for 48 wks.) in 15 adult and pediatric patients with plasminogen deficiency type 1 The therapy showed at least 50% improvement in lesions in 11 patients with lesions at baseline and absence of recurrent or …

ProMetic’ Ryplazim (plasminogen) Receives the US FDA’s Approval as First Treatment for Patients with Plasminogen Deficiency Read More »

Dermavant Submits NDA to FDA for Tapinarof Cream to Treat Plaque Psoriasis

Shots: The submission is based on data from the P-III PSOARING 1 & PSOARING as well as interim results from PSOARING 3 long-term safety study 2 evaluating the safety and efficacy of tapinarof vs vehicle in patients with plaque psoriasis Results: Improvement in PGA scores of 0 or 1 with a minimum 2-grade improvement from …

Dermavant Submits NDA to FDA for Tapinarof Cream to Treat Plaque Psoriasis Read More »

Innovent and Lilly’s Tyvyt (sintilimab injection) + Gemcitabine and Platinum CT Receive NMPA’s Approval as 1L Therapy for Squamous Non-Small Cell Lung Cancer

Shots: The approval is based on P-III ORIENT-12 trial evaluating Tyvyt (sintilimab injection) or PBO + Gemzar (gemcitabine) & platinum CT in 357 patients in a ratio (1:1) as 1L therapy for advanced or metastatic sqNSCLC Results: Improvement in PFS & OS, m-PFS (5.5 vs 4.9 mos.) as assessed by IRRC & (6.7 vs 4.9 …

Innovent and Lilly’s Tyvyt (sintilimab injection) + Gemcitabine and Platinum CT Receive NMPA’s Approval as 1L Therapy for Squamous Non-Small Cell Lung Cancer Read More »

BMS’ Opdivo (nivolumab) + Yervoy (ipilimumab) Receive EC’s Approval for 1L Treatment for Unresectable Malignant Pleural Mesothelioma

Shots: The approval is based on P-III CheckMate -743 study assessing Opdivo (3 mg/kg, q2w) + Yervoy (1 mg/kg, q6w) vs CT in patients with previously untreated MPM The trial met its 1EP i.e improvement in OS (26% reduction in risk of death) m-OS (18.1 vs 14.1 mos.), ORR (40% vs 43%), DoR (11.0 vs …

BMS’ Opdivo (nivolumab) + Yervoy (ipilimumab) Receive EC’s Approval for 1L Treatment for Unresectable Malignant Pleural Mesothelioma Read More »

Janssen’s Teclistamab Receives the US FDA’s Breakthrough Therapy Designation for Relapsed or Refractory Multiple Myeloma

Shots: The FDA’s BT and EMA’s PRIME designations are based on data from the P-I MajesTEC-1 study evaluating the safety and efficacy of teclistamab (Ab targeting BCMA and CD3) in adults with r/rMM. The findings will be presented at ASCO 2021 In preclinical studies, the therapy kills myeloma cell lines and bone marrow-derived myeloma cells …

Janssen’s Teclistamab Receives the US FDA’s Breakthrough Therapy Designation for Relapsed or Refractory Multiple Myeloma Read More »

Novartis’ Cosentyx (secukinumab) Receives the US FDA’s Approval for the Treatment of Moderate to Severe Plaque Psoriasis

Shots: The approval is based on two P-III studies evaluating Cosentyx (75 mg/ 150 mg) vs PBO in children aged 6 to <18 yrs with plaque psoriasis The first study demonstrates reduction in psoriasis severity @12 wks., PASI 75 response for 75mg and 150mg (55%, 86% vs 10%, 19%), achieved clear or almost clear skin …

Novartis’ Cosentyx (secukinumab) Receives the US FDA’s Approval for the Treatment of Moderate to Severe Plaque Psoriasis Read More »

Biohaven’s Nurtec ODT (rimegepant) Receives the US FDA’s Approval for Treatment of Migraine

Shots: The approval is based on the P-III OLE trial involves assessing Nurtec ODT (rimegepant, 75 mg) vs PBO in patients with episodic migraine who experience less than 15 headache days/mos. The 1EPs & 2EPs of the trial demonstrate a reduction in monthly migraine days by 4.3 days/mos. after 3mos. treatment, 50% patients reported at …

Biohaven’s Nurtec ODT (rimegepant) Receives the US FDA’s Approval for Treatment of Migraine Read More »

BMS’ Abecma (idecabtagene vicleucel) Receives the Health Canada’s Approval as the First Anti-BCMA CAR T Cell Therapy for Relapsed and Refractory Multiple Myeloma

Shots: The conditional approval is based on the results of the P-II KarMMa study involves assessing Abecma in 122 patients with RRMM prior treated with 3L+ therapy including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 Ab and who are refractory to their last treatment Results: @median follow-up of 13.3 mos., ORR (74%); CR …

BMS’ Abecma (idecabtagene vicleucel) Receives the Health Canada’s Approval as the First Anti-BCMA CAR T Cell Therapy for Relapsed and Refractory Multiple Myeloma Read More »

Ipsen Reports the US FDA’s Acceptance of NDA for Palovarotene to Treat Fibrodysplasia Ossificans Progressiva

Shots: The NDA submission is based on data from the ongoing P-III MOVE trial evaluating the efficacy and safety of palovarotene (chronic/flare-up dosing regimen) in patients with FOP The post hoc analyses of 1EPs from the P-III MOVE trial demonstrated a 62% reduction in mean annualized new HO volume in patients with palovarotene vs untreated …

Ipsen Reports the US FDA’s Acceptance of NDA for Palovarotene to Treat Fibrodysplasia Ossificans Progressiva Read More »

Novo Nordisk Resubmits Label Expansion Application to the US FDA for Semaglutide (2.0mg)

Shots: The company has announced the resubmission of a label expansion application to the US FDA for the existing marketing authorization for Ozempic to introduce a new dose of 2.0mg for the Treatment of T2D The resubmission follows the Refusal to File letter received by the FDA on 22 March 2021. The standard review time …

Novo Nordisk Resubmits Label Expansion Application to the US FDA for Semaglutide (2.0mg) Read More »

BridgeBio and Helsinn’s Truseltiq (infigratinib) Receive the US FDA’s Approval for Cholangiocarcinoma

Shots: The approval is based on a P-II study assessing Truseltiq (125mg, qd, for 21 days of 28-day cycles) in 108 patients who had undergone at least one prior treatment for advanced CCA. Out of 108, 107 had Stage IV CCA Results: ORR (23%); mDOR (5mos.); presented at ASCO 2021. Additional marketing applications for Truseltiq …

BridgeBio and Helsinn’s Truseltiq (infigratinib) Receive the US FDA’s Approval for Cholangiocarcinoma Read More »

Pfizer and BioNTech Receive EC’s Conditional Marketing Approval for the COVID-19 Vaccine in Adolescents

Shots: The approval is based on a P-III study assessing Comirnaty (two 30μg doses) in 2,260 participants aged 12-15yrs. and showed 100% efficacy in participants with/out prior SARS-CoV-2 infection, robust Ab responses and was well tolerated The approval follows CHMP’s positive opinion to authorize the vaccine in this group. The extended indication for the CMA …

Pfizer and BioNTech Receive EC’s Conditional Marketing Approval for the COVID-19 Vaccine in Adolescents Read More »

Amgen’s Lumakras (sotorasib) Receives the US FDA’s Approval as the First KRAS Blocking Cancer Therapy

Shots: The approval is based on the CodeBreaK 100 trial evaluating the efficacy & tolerability of Lumakras (960mg, qd) in 124 patients with KRAS G12C mutation-positive NSCLC whose disease had progressed after prior treatment with immunotherapy or CT Results: ORR (36%); DCR (81%); mDoR (10 mos.); durable response with a positive benefit-risk profile, 9% showed …

Amgen’s Lumakras (sotorasib) Receives the US FDA’s Approval as the First KRAS Blocking Cancer Therapy Read More »

Seelos’ SLS-005 (Trehalose) Receives EC’s Orphan Drug Designation to Treat Amyotrophic Lateral Sclerosis

Shots: EMA’s COMP has granted ODD to SLS-005 for the treatment of amyotrophic lateral sclerosis Under ODD, Seelos will get benefit from several incentives such as protocol assistance, reduced regulatory fees and market exclusivity Trehalose is a low molecular weight disaccharide (0.342 kDa) that crosses the BBB, stabilizes proteins and activates autophagy and has received …

Seelos’ SLS-005 (Trehalose) Receives EC’s Orphan Drug Designation to Treat Amyotrophic Lateral Sclerosis Read More »

The US FDA’s EMDAC Supports Provention Bio’s Teplizumab in Delaying Diabetes

Shots: EMDAC has voted 10-7, confirming that the benefits of teplizumab outweigh the risks supporting the approval to delay T1D Mellitus The recommendation is based on a pivotal TN-10 study in which a single 14-day course of teplizumab delayed insulin-dependent, clinical-stage disease by a median of at least 2yrs. in pre symptomatic patients with stage …

The US FDA’s EMDAC Supports Provention Bio’s Teplizumab in Delaying Diabetes Read More »

AstraZeneca’s Tagrisso (osimertinib) Receives EU’s Approval for the Adjuvant Treatment of Patients with Early-Stage EGFR-Mutated Lung Cancer

Shots: The approval is based on P-III ADAURA trial assessing Tagrisso (80mg, qd, PO) vs PBO in 682 patients with stage IB, II, IIIA EGFRm NSCLC following complete tumor resection and adjuvant CT as indicated for 3 yrs. or until disease recurrence Results: Improvement in DFS in all primary analysis population in Stage II and …

AstraZeneca’s Tagrisso (osimertinib) Receives EU’s Approval for the Adjuvant Treatment of Patients with Early-Stage EGFR-Mutated Lung Cancer Read More »

BMS’ Zeposia (ozanimod) Receives the US FDA’s Approval for Ulcerative Colitis

Shots: The approval is based on a P-III True North trial assess Zeposia (0.92 mg) vs PBO in an adult with mod. to sev. active UC The trial met its 1EPs & 2EPs i.e Zeposia as an induction & maintenance therapy demonstrates clinical remission (18% vs 6% & 37% vs 19%), clinical response (48% vs …

BMS’ Zeposia (ozanimod) Receives the US FDA’s Approval for Ulcerative Colitis Read More »

ONO and BMS’ Opdivo + Yervoy Receive Approval for 1L Treatment for Unresectable Advanced or Recurrent Malignant Pleural Mesothelioma

Shots: The approval is based on P-III CheckMate -743 study assessing Opdivo + Yervoy vs SOC Pt. based CT in patients with previously untreated unresectable malignant pleural mesothelioma The combination regimen has demonstrated significant extension of OS vs CT. The safety profile of combination treatment was consistent with those previously reported in the studies for …

ONO and BMS’ Opdivo + Yervoy Receive Approval for 1L Treatment for Unresectable Advanced or Recurrent Malignant Pleural Mesothelioma Read More »

Eli Lilly to Initiate P-I Study of LY3819469 for Cardiometabolic Diseases

Shots: Eli Lilly and Dicerna have reported the US FDA’s IND acceptance of LY3819469 to treat cardiometabolic diseases. The IND acceptance triggers $10M as a milestone to Dicerna under the companies’ 2018 research collaboration and licensing agreement LY3819469 is an investigational GalXC RNAi candidate targeting the LPA gene and is the second clinical-stage candidate to …

Eli Lilly to Initiate P-I Study of LY3819469 for Cardiometabolic Diseases Read More »

Pfizer and Myovant’s Myfembree Receive the US FDA’s Approval for Heavy Menstrual Bleeding Associated with Uterine Fibroids

Shots: The approval is based on efficacy and safety data from the P-III LIBERTY 1 & 2 studies assessing Myfembree (relugolix 40 mg, estradiol 1 mg & norethindrone acetate 0.5 mg) vs PBO for heavy menstrual bleeding associated with uterine fibroids in premenopausal women with a treatment duration for up to 2yrs. The P-III studies …

Pfizer and Myovant’s Myfembree Receive the US FDA’s Approval for Heavy Menstrual Bleeding Associated with Uterine Fibroids Read More »

GSK and Vir Receive the US FDA’s EUA for Sotrovimab (VIR-7831) to Treat COVID-19 in Adults and Pediatric Patients

Shots: The US FDA has granted EUA for sotrovimab for mild to mod. COVID-19 patients aged ≥12yrs. who are at high risk for progression to severe COVID-19 including hospitalization for >24hrs. or death The EUA is based on an interim analysis from the P-III COMET-ICE trial that demonstrated an 85% reduction in hospitalization or death. …

GSK and Vir Receive the US FDA’s EUA for Sotrovimab (VIR-7831) to Treat COVID-19 in Adults and Pediatric Patients Read More »

TG Therapeutics Reports the US FDA’s Acceptance of BLA for Ublituximab + Ukoniq (umbralisib) to Treat Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma

Shots: The BLA submission is based on P-III UNITY-CLL trial evaluating ublituximab + Ukoniq (U2) vs obinutuzumab + chlorambucil in a ratio (1:1) in 420 patients with treatment-naive and r/r CLL. The 1EP of the study was PFS The trial is being conducted under a SPA agreement with the US FDA. The FDA also notified …

TG Therapeutics Reports the US FDA’s Acceptance of BLA for Ublituximab + Ukoniq (umbralisib) to Treat Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma Read More »

AbbVie’s Venclyxto Receives the EC’s Approval for Patients with Newly Diagnosed Acute Myeloid Leukemia who are Ineligible for Intensive Chemotherapy

Shots: The approval is based on P-III VIALE-A and P-Ib M14-358 studies. The P-III study involves assessing Venclyxto + azacitidine in patients with newly diagnosed AML who were ineligible for intensive CT and demonstrated OS (14.7mos. vs 9.6mos.), CCCR (66.4% vs 28.3%), CR (36.7% vs 17.9%) The P-Ib M14-358 trial involves assessing in Venclyxto + …

AbbVie’s Venclyxto Receives the EC’s Approval for Patients with Newly Diagnosed Acute Myeloid Leukemia who are Ineligible for Intensive Chemotherapy Read More »

Boehringer Ingelheim’s BI 425809 Receives the US FDA’s Breakthrough Therapy Designation for Cognitive Impairment Associated with Schizophrenia

Shots: The BTD is based on P-II 1346.9 clinical trial, which demonstrated that BI 425809 improved cognition in adult patients with schizophrenia Moreover, the company has initiated the P-III CONNEX program assessing the safety and efficacy of BI 425809 vs PBO for improving cognition in adults with schizophrenia over a 26wks. treatment period Additionally, Boehringer …

Boehringer Ingelheim’s BI 425809 Receives the US FDA’s Breakthrough Therapy Designation for Cognitive Impairment Associated with Schizophrenia Read More »

Bayer Seeks Approval of Larotrectinib for Advanced Solid Tumors in China

Shots: The NDA submission is based on P-I study of adult patients, the P-II NAVIGATE trial in adult and adolescent patients and the P- I/II pediatric SCOUT trial. These trials evaluated Larotrectinib in 20+ different histologies of solid tumors with NTRK fusion The therapy is already approved in several countries under the brand name Vitrakvi, …

Bayer Seeks Approval of Larotrectinib for Advanced Solid Tumors in China Read More »

Regeneron Receives CHMP’s Positive Opinion Recommending the Approval of Libtayo (cemiplimab) for NSCLC and BCC

Shots: The CHMP recommended the approval of Libtayo as a 1L treatment for adults with NSCLC expressing PD-L1 in ≥50% of tumor cells with no EGFR, ALK or ROS1 aberrations, based on the P-III EMPOWER-Lung 1 study The positive opinion of Libtayo in LA and mBCC is based on P-II EMPOWER-BCC 1 study in patients …

Regeneron Receives CHMP’s Positive Opinion Recommending the Approval of Libtayo (cemiplimab) for NSCLC and BCC Read More »

Janssen’s Rybrevant (amivantamab-vmjw) Receives the US FDA’s Approval as the First Targeted Treatment for Patients with NSCLC with EGFR Exon 20 Insertion Mutations

Shots: The US FDA has granted accelerated approval which is based on P-I CHRYSALIS study assessing Rybrevant as a monothx. in patients metastatic non-small cell lung cancer with epidermal growth factor receptor exon 20 insertion mutations whose disease has progressed on or after platinum-based CT The FDA has also approved Guardant Health’s Guardant360 CDx liquid …

Janssen’s Rybrevant (amivantamab-vmjw) Receives the US FDA’s Approval as the First Targeted Treatment for Patients with NSCLC with EGFR Exon 20 Insertion Mutations Read More »

BMS Receive the CHMP’s Positive Opinion Recommending Approval of Opdivo (nivolumab) + Yervoy (ipilimumab) for dMMR or MSI-H mCRC After Prior Chemotherapy

Shots: The opinion is based on P-II CheckMate -142 study assessing Opdivo + Yervoy in patients with mismatch repair deficient or microsatellite instability–high metastatic colorectal cancer whose disease had progressed during or after prior treatment with CT If approved, Opdivo in combination with Yervoy will be the first dual immunotherapy approved in the EU in …

BMS Receive the CHMP’s Positive Opinion Recommending Approval of Opdivo (nivolumab) + Yervoy (ipilimumab) for dMMR or MSI-H mCRC After Prior Chemotherapy Read More »

Takeda Reports the US FDA’s Acceptance and Priority Review of Maribavir’s NDA for Post-Transplant CMV Infection

Shots: The NDA is based on P-III TAK-620-303 (SOLSTICE) trial assessing Maribavir vs IAT in HCT and SOT recipients with CMV infection refractory, with/ out resistance, to one or a combination of the conventional antiviral therapies Patients underwent a 2wks. screening period, followed by randomization (2:1) to maribavir (400 mg) or IAT for an 8wks. …

Takeda Reports the US FDA’s Acceptance and Priority Review of Maribavir’s NDA for Post-Transplant CMV Infection Read More »

AstraZeneca’s Vaxzevria (AZD1222) Receives Approval for Emergency Use in Japan to Treat COVID-19

Shots: MHLW has granted EUA for Vaxzevria (ChAdOx1-S [Recombinant]) for active immunization of individuals aged ≥18yrs. to prevent COVID-19. The EUA is based on P-III data from the Oxford University-led clinical trial in the UK, Brazil and South Africa, and a P-I/II trial in Japan PMDA recommends the use of Vaxzevria should be limited to …

AstraZeneca’s Vaxzevria (AZD1222) Receives Approval for Emergency Use in Japan to Treat COVID-19 Read More »

Moderna’s COVID-19 Vaccine Receives Approval for Emergency Use in Japan

Shots: MHLW has granted special approval under article 14-3 of the PMDA for emergency use of Moderna’s mRNA COVID-19 vaccine based on Takeda’s P-I/II immunogenicity and safety clinical trial The interim analysis showed that binding Ab and neutralizing Ab titers were elevated @28days after the second dose in 100% of people vaccinated with two 0.5ml …

Moderna’s COVID-19 Vaccine Receives Approval for Emergency Use in Japan Read More »

BioXcel Reports the US FDA’s Acceptance of BXCL501’s NDA for the Acute Treatment of Agitation Associated with Schizophrenia and Bipolar Disorders I and II

Shots: The NDA submission is based on P-III studies (SERENITY I & II) assessing BXCL501 (120 & 180mcg doses) vs PBO for the acute treatment of agitation associated with schizophrenia and bipolar disorders I and II, respectively Results: BXCL501 met its 1EPs & 2EPs in both the studies, demonstrating rapid, and durable improvements from baseline …

BioXcel Reports the US FDA’s Acceptance of BXCL501’s NDA for the Acute Treatment of Agitation Associated with Schizophrenia and Bipolar Disorders I and II Read More »

Roche Receives the US FDA’s EUA for cobas SARS-CoV-2 Test to Suspect COVID-19

Shots: Roche’s cobas SARS-CoV-2 test to be use on high-throughput cobas 6800/8800 systems has received the US FDA’s EUA for testing individuals without symptoms or reasons to control the spread of COVID-19 The authorization expands SARS-CoV-2 testing to include people without symptoms and applies to pooled samples containing up to and including 6 individual samples …

Roche Receives the US FDA’s EUA for cobas SARS-CoV-2 Test to Suspect COVID-19 Read More »

Eli Lilly and Innovent Report the US FDA’s Acceptance of Sintilimab + Chemotherapy for 1L Treatment for Non-Squamous NSCLC

Shots: The submission is based on a P-III ORIENT-11 study assessing sintilimab (200mg) + pemetrexed and platinum CT vs PBO + CT as a 1L treatment for 397 patients in a ratio (2:1) with advanced or metastatic nonsq. NSCLC, with no sensitizing EGFR mutations or ALK rearrangements The anticipated PDUFA date for this application is …

Eli Lilly and Innovent Report the US FDA’s Acceptance of Sintilimab + Chemotherapy for 1L Treatment for Non-Squamous NSCLC Read More »

Abbott Receives CE Mark for Next-Generation Navitor TAVI System to Treat Aortic Stenosis

Shots: Abbott has received CE Mark for Navitor making the minimally invasive device available for people in Europe with severe aortic stenosis who are at high or extreme surgical risk Navitor uses Abbott’s FlexNav delivery system along with a unique design for preventing blood leaking around the valve to provide a less invasive alternative to …

Abbott Receives CE Mark for Next-Generation Navitor TAVI System to Treat Aortic Stenosis Read More »

Apellis’ Empaveli (pegcetacoplan) Receives the US FDA’s Approval for Paroxysmal Nocturnal Hemoglobinuria

Shots: The approval is based on P-III head-to-head PEGASUS study assessing Empavel vs Soliris (eculizumab) in 80 adults with PNH. Participants should be Soliris stable for at least 3mos. with a Hgb level of <10.5 g/dL at the screening visit Results: The study met its 1EPs demonstrating superiority to Soliris for the change from baseline …

Apellis’ Empaveli (pegcetacoplan) Receives the US FDA’s Approval for Paroxysmal Nocturnal Hemoglobinuria Read More »

Heron Therapeutics’ ZYNRELEF (HTX-011) Receives the US FDA’s Approval for the Management of Postoperative Pain

Shots: The US FDA approved Zynrelef (bupivacaine and meloxicam) extended-release solution for postsurgical analgesia in patients with soft tissue or periarticular instillation for 72 hrs after a bunionectomy, open inguinal herniorrhaphy and total knee arthroplasty The approval is based on data from the P-III studies assessing Zynrelef vs bupivacaine solution in 1000 patients which demonstrated …

Heron Therapeutics’ ZYNRELEF (HTX-011) Receives the US FDA’s Approval for the Management of Postoperative Pain Read More »

Fulcrum Therapeutics’ Losmapimod Receives the US FDA’s Fast Track Designation to Treat Facioscapulohumeral Muscular Dystrophy

Shots: The US FDA has granted FTD to losmapimod for the treatment of FSHD while the therapy has previously received ODD for FSHD The company will present full data from the P-IIb ReDUX4 trial assessing losmapimod vs PBO in patients with FSHD at FSHD on June 24-25, 2021 that include 1EP results i.e., reduction from …

Fulcrum Therapeutics’ Losmapimod Receives the US FDA’s Fast Track Designation to Treat Facioscapulohumeral Muscular Dystrophy Read More »

Antengene Receives IND Approval for P-III SIENDO Trial of XPOVIO (Selinexor) to Treat Advanced or Recurrent Endometrial Cancer in China

Shots: The NMPA has approved the IND application for the P- III SIENDO trial to evaluate the safety and efficacy of selinexor for advanced or recurrent endometrial cancer. The trial is being conducted over 80 centers across North America, Europe, and Asia and the results are expected in H2’2021 Antengene holds the exclusive development and …

Antengene Receives IND Approval for P-III SIENDO Trial of XPOVIO (Selinexor) to Treat Advanced or Recurrent Endometrial Cancer in China Read More »

Supernus’ Qelbree (SPN-812) Receives the US FDA’s Approval for the Treatment of ADHD

Shots: The approval is based on data from an extensive development program that consists of four P-III clinical trials evaluating Qelbree in 1000+ pediatric patients aged 6 to 17 yrs. with ADHD The program demonstrated the proven efficacy and a tolerable safety profile. In Dec’2020, the company reported the results from the P- III trial …

Supernus’ Qelbree (SPN-812) Receives the US FDA’s Approval for the Treatment of ADHD Read More »

AstraZeneca and Amgen Submit BLA to US FDA for Tezepelumab to Treat Severe Asthma

Shots: The submission is based on PATHFINDER clinical program including P-III NAVIGATOR that involves assessing Tezepelumab + SOC vs PBO + SOC in 1061 adults (18–80yrs.) & adolescents (12–17yrs.) with severe and uncontrolled asthma The study demonstrated 56% reduction in AAER @52wks. in the overall population. The study also met 1EPs in the subgroup of …

AstraZeneca and Amgen Submit BLA to US FDA for Tezepelumab to Treat Severe Asthma Read More »

Pfizer and BioNTech Receive the US FDA’s EMA for COVID-19 Vaccine in Adolescents

Shots: The FDA has expanded the EUA for Pfizer’s COVID-19 vaccine in adolescents based on the data from the P- III trial in 2,260 participants aged 12-15yrs. which showed 100% efficacy and was well-tolerated. The submission of data in children aged 6mos.- 2yrs. are expected in Q4’21 The companies have submitted the data in adolescents …

Pfizer and BioNTech Receive the US FDA’s EMA for COVID-19 Vaccine in Adolescents Read More »

Pfizer and BioNTech Initiate Rolling Submission of BLA for the US FDA’s Approval of their COVID-19 Vaccine

Shots: The companies initiate the BLA with the FDA for approval of the COVID-19 vaccine in patients aged ≥16yrs. The data supporting the BLA will be submitted on a rolling basis over the coming wks., with a request for Priority Review Additionally, the companies will submit the required manufacturing & facility data for licensure to …

Pfizer and BioNTech Initiate Rolling Submission of BLA for the US FDA’s Approval of their COVID-19 Vaccine Read More »

EU starts rolling review of GSK’s antibody for COVID-19

The EMA has begun a rolling review of a COVID-19 antibody developed by GlaxoSmithKline and Vir BioTech which could become the fourth drug of its type cleared for early use in the EU.  Sotrovimab (VIR-7831) – also known as GSK4182136 – is being assessed as a treatment for patients with SARS-CoV-2 infection aged 12 and …

EU starts rolling review of GSK’s antibody for COVID-19 Read More »

GSK and Vir Report Initiation of EMA Rolling Review of VIR-7831 (sotrovimab) for the Early Treatment of COVID-19

Shots: The rolling review will assess sotrovimab in adults and adolescents (aged 12yrs.) with COVID-19 who do not require oxygen supplementation and who are at risk of progressing to severe COVID-19. The rolling review will support a formal MAA EMA’s CHMP will review the data and the decision to start the rolling review is based …

GSK and Vir Report Initiation of EMA Rolling Review of VIR-7831 (sotrovimab) for the Early Treatment of COVID-19 Read More »

Merck and Eisai Receive the US FDA‘s Priority Review for Keytruda (pembrolizumab) + Lenvima (lenvatinib) Applications to Treat Advanced Renal Cell Carcinoma and Endometrial Carcinoma

Shots: The 1st sNDA and sBLA is based on results from the P-III CLEAR Study evaluating Lenvima + Keytruda or in combination with everolimus vs sunitinib in a ratio (1:1:1) in 1,069 patients with advanced RCC, demonstrated improved PFS, OS, and ORR The 2nd application is based on results from the P-III KEYNOTE-775/Study 309 trial …

Merck and Eisai Receive the US FDA‘s Priority Review for Keytruda (pembrolizumab) + Lenvima (lenvatinib) Applications to Treat Advanced Renal Cell Carcinoma and Endometrial Carcinoma Read More »

Amgen Reports the US FDA’s Acceptance of sNDA for Review of Otezla (apremilast) to Treat Mild-To-Moderate Plaque Psoriasis

Shots: The acceptance is based on a P-III ADVANCE trial assessing the efficacy of Otezla (30 mg, bid) vs PBO in 595 patients in a ratio (1:1) with mild-to-moderate plaque psoriasis for the first 16wks. All patients then received Otezla during an OLE phase through week 32. Results: @16wks., improvement in its 1EPs of sPGA …

Amgen Reports the US FDA’s Acceptance of sNDA for Review of Otezla (apremilast) to Treat Mild-To-Moderate Plaque Psoriasis Read More »

GSK’s Benlysta (belimumab) Receives EC’s Approval for the Treatment of Active Lupus Nephritis

Shots: The MAA is based on a P-III BLISS-LN study assessing the efficacy and safety of belimumab (10 mg/kg, IV) + standard therapy vs PBO + standard therapy in 448 patients with active lupus nephritis for 104 wks. The study met its 1EPs i.e patients who achieved PERR @2yrs. (43% vs 32%) and has achieved …

GSK’s Benlysta (belimumab) Receives EC’s Approval for the Treatment of Active Lupus Nephritis Read More »

Roche’s Tecentriq Receives EC’s Approval as 1L Treatment for Metastatic Non-Small Cell Lung Cancer

Shots: The approval is based on P-III IMpower110 study assessing the efficacy and safety of Tecentriq vs CT in 572 PD-L1-selected, CT-naïve participants in a ratio (1:1) with stage IV non-squamous or squamous NSCLC Results: improvement in OS in people with high PD-L1 expression (20.2 vs 13.1mos.), safety was consistent with its known safety profile …

Roche’s Tecentriq Receives EC’s Approval as 1L Treatment for Metastatic Non-Small Cell Lung Cancer Read More »

AstraZeneca’s Farxiga (dapagliflozin) Receives the US FDA’s Approval for Chronic Kidney Disease

Shots: The approval is based on P-III DAPA-CKD trial assessing Farxiga (qd, 10 mg) + SOC vs PBO in 4304 patients with CKD stages 2-4 and elevated urinary albumin excretion, with/out T2D Results: 39% reduction in relative risk of worsening of renal function or risk of CV or renal death, ARR (5.3%) @2.4yrs., and reduction …

AstraZeneca’s Farxiga (dapagliflozin) Receives the US FDA’s Approval for Chronic Kidney Disease Read More »

BMS Reports the US FDA’s Acceptance of Opdivo’s sBLA for Priority Review to Treat Muscle Invasive Urothelial Carcinoma

Shots: The filing is based on results from the P-III CheckMate -274 trial evaluating Opdivo (240mg, q2w for up to 1 yrs.) vs PBO in a ratio (1:1) in 709 patients with muscle-invasive urothelial cancer who are at a high risk of recurrence after radical surgery The study demonstrated the improvement in DFS regardless of …

BMS Reports the US FDA’s Acceptance of Opdivo’s sBLA for Priority Review to Treat Muscle Invasive Urothelial Carcinoma Read More »

Janssen Reports MAA Submission to EMA for Cilta-cel to Treat Relapsed/ Refractory Multiple Myeloma

Shots: The application is based on P-Ib/II CARTITUDE-1 study evaluating cilta-cel in adults with r/r MM who have received at least prior 3L therapies across the US, EU, China, and Japan The 1EP of the P-Ib study is to characterize the safety and confirm the dose of the therapy while the primary objective of P-II …

Janssen Reports MAA Submission to EMA for Cilta-cel to Treat Relapsed/ Refractory Multiple Myeloma Read More »

Y-mAbs Reports MAA Submission to EMA for Omburtamab to Treat Pediatric Patients with CNS/Leptomeningeal Metastasis from Neuroblastoma

Shots: The company has submitted MAA for Omburtamab to EMA for the treatment of pediatric patients with CNS/leptomeningeal metastasis from neuroblastoma The company continues to advance the Omburtamab program addressing the unmet medical need globally and work closely with FDA to resubmit the BLA in Q2 or Q3’21 Y-mAbs get an exclusive license from MSK …

Y-mAbs Reports MAA Submission to EMA for Omburtamab to Treat Pediatric Patients with CNS/Leptomeningeal Metastasis from Neuroblastoma Read More »

The US FDA Grants Priority Review to Takeda’s Mobocertinib (TAK-788) for EGFR Exon20 Insertion+ Metastatic Non-Small Cell Lung Cancer

Shots: The US FDA has granted PR for mobocertinib’s NDA to treat adult patients with EGFR Exon20 insertion+ mNSCLC, as detected by an FDA-approved test, who have received prior Pt-based CT. The anticipated PDUFA date is Oct 26, 2021 The NDA is based on a P-I/II study assessing mobocertinib (PO) in patients with mNSCLC. The …

The US FDA Grants Priority Review to Takeda’s Mobocertinib (TAK-788) for EGFR Exon20 Insertion+ Metastatic Non-Small Cell Lung Cancer Read More »

Glenmark’s Ryaltris Nasal Spray Receives EC’s Approval as 1L Treatment for Allergic Rhinitis

Shots: Glenmark is concluding the final, national phase of its MAA process to enable launch of its innovative nasal spray, Ryaltris (olopatadine hydrochloride, 665mcg and mometasone furoate, 25mcg), in 17 countries in the EU Menarini will lead the commercialization in France, Italy, Spain, and the Balkan region under its collaboration with Glenmark signed in 2020. …

Glenmark’s Ryaltris Nasal Spray Receives EC’s Approval as 1L Treatment for Allergic Rhinitis Read More »

AstraZeneca’s Tagrisso (osimertinib) Receives EU’s CHMP Positive Opinion for the Adjuvant Treatment of Patients with Early-Stage EGFR-Mutated Lung Cancer

Shots: The recommendation is based P-III ADAURA trial assessing Tagrisso (80mg, qd, PO) vs PBO in 682 patients with stage IB, II, IIIA EGFRm NSCLC following complete tumor resection and adjuvant CT as indicated for 3 yrs. or until disease recurrence The study showed improvement in DFS in all primary analysis population of patients with …

AstraZeneca’s Tagrisso (osimertinib) Receives EU’s CHMP Positive Opinion for the Adjuvant Treatment of Patients with Early-Stage EGFR-Mutated Lung Cancer Read More »

AstraZeneca and MSD’s Selumetinib Receive EU’s CHMP Positive Opinion as the First Therapy for Pediatric Patients with Neurofibromatosis Type 1 Plexiform Neurofibromas

Shots: EMA’s CHMP has recommended conditional marketing authorization of Selumetinib in the EU. The recommendation is based on the results from the P-I/II SPRINT Stratum 1 trial evaluating selumetinib as a monothx (PO, bid) in pediatric patients aged ≥3yrs. with NF1 and symptomatic, inoperable PNs The trial showed 66% ORR which is defined as the …

AstraZeneca and MSD’s Selumetinib Receive EU’s CHMP Positive Opinion as the First Therapy for Pediatric Patients with Neurofibromatosis Type 1 Plexiform Neurofibromas Read More »

GSK’s Jemperli (dostarlimab) Receives the EC’s Approval for Recurrent or Advanced dMMR/MSI-H Endometrial Cancer

Shots: The EC has granted conditional approval for Jemperli in women with dMMR/ MSI-H endometrial cancer that has progressed on or following prior treatment with a Pt.- containing regimen AnaptysBio has received $10M as milestones upon the EC’s approval and will receive $35M as regulatory milestones in the US & EU, $165M as sales milestones …

GSK’s Jemperli (dostarlimab) Receives the EC’s Approval for Recurrent or Advanced dMMR/MSI-H Endometrial Cancer Read More »

BMS’ Opdivo + Yervoy and Onureg Receive EC’s CHMP Positive Opinion for MPM and AML

Shots: The CHMP has recommended the approval of Opdivo (nivolumab) + Yervoy (ipilimumab) as 1L treatment for unresectable MPM and is based P-III CheckMate -743 trial that met its 1EPs of OS The opinion for Onureg is based P-III QUAZAR AML-001 study assessing Onureg (300mg, qd) vs PBO in 472 patients in a ratio (1:1) …

BMS’ Opdivo + Yervoy and Onureg Receive EC’s CHMP Positive Opinion for MPM and AML Read More »

Gilead Reports NDA Submission of Filgotinib to PMDA for Ulcerative Colitis in Japan

Shots: The submission is based on P-IIb/III SELECTION study evaluating the efficacy and safety of filgotinib (200mg, qd) vs PBO in patients with mod. to sev. active UC who had an inadequate response or were intolerant to either conventional therapy or a biological agent Results: patients achieved clinical remission @10wks., maintained remission @ 58 wks. …

Gilead Reports NDA Submission of Filgotinib to PMDA for Ulcerative Colitis in Japan Read More »

AbbVie’s Humira (adalimumab) Receives Health Canada’s Approval for Pediatric Patients with Active Ulcerative Colitis

Shots: The approval is based on P-III ENVISION I study that involves assessing the efficacy, safety, and PK of Humira (SC) in pediatric patients aged 4-17yrs. with mod. to sev. UC The study met its co-1EPs of clinical remission @8wks, patients who responded @8wks, achieved clinical remission @52wks., no new safety signals were identified Humira …

AbbVie’s Humira (adalimumab) Receives Health Canada’s Approval for Pediatric Patients with Active Ulcerative Colitis Read More »

GSK’s Jemperli (dostarlimab-gxly) Receives the US FDA’s Approval for dMMR Endometrial Cancer

Shots: The US FDA has approved the BLA filing for Jemperli for adult patients with dMMR recurrent or advanced solid tumors endometrial cancer, as determined by an FDA-approved test, that has progressed on or following prior treatment with a Pt.-containing regimen AnaptysBio has received $20M as milestones upon the FDA’s approval and will receive $45M …

GSK’s Jemperli (dostarlimab-gxly) Receives the US FDA’s Approval for dMMR Endometrial Cancer Read More »

Roche Receives the US FDA’s Approval for First CDx to Identify Endometrial Cancer Patients Eligible for Immunotherapy

Shots: The US FDA has approved Ventana MMR RxDx panel to identify endometrial cancer patients who are eligible for treatment with GSK’s Jemperli (monothx.) that has received the US FDA’s approval on Apr 22, 2021 The CDx provides clinicians with a standardized testing option that uses a comprehensive panel of DNA mismatch repair (MMR) biomarkers …

Roche Receives the US FDA’s Approval for First CDx to Identify Endometrial Cancer Patients Eligible for Immunotherapy Read More »

LEXEO Therapeutics Receives the US FDA’s Fast Track Designation for LX1001 to Treat APOE4 Associated Alzheimer Disease

Shots: The US FDA has granted FT Designation to LX1001 for the treatment of APOE4 associated AD. The designation evaluates to facilitate the development and expedite the review of drug The company has initiated P-I trial to assess the safety and toxicity of LX1001 as a potential one-time treatment for early-stage AD patients with mild …

LEXEO Therapeutics Receives the US FDA’s Fast Track Designation for LX1001 to Treat APOE4 Associated Alzheimer Disease Read More »

GW Pharmaceuticals’ Epidyolex (cannabidiol) Receives EC’s Approval for the Treatment of Seizures Associated with Tuberous Sclerosis Complex

Shots: The approval is based on P-III study evaluating cannabidiol (25 mg/kg/day) vs PBO in patients aged ≥2yrs. as an adjunctive treatment of seizures associated with TSC The study met its 1EPs i.e reduction in seizure frequency (49% vs 27%). The 2EPs supports the effects on 1EPs and safety profile was consistent with findings from …

GW Pharmaceuticals’ Epidyolex (cannabidiol) Receives EC’s Approval for the Treatment of Seizures Associated with Tuberous Sclerosis Complex Read More »

Janssen’s Darzalex (daratumumab, SC) Combination Regimen Receives Health Canada’s Approval for Patients with Newly Diagnosed Light Chain Amyloidosis

Shots: The approval is based on results from the P-III ANDROMEDA (AMY3001) study involves assessing the safety and efficacy of Darzalex (1800mg, SC, qw) + bortezomib, cyclophosphamide, and dexamethasone (VCd) vs VCd alone in 388 patients with newly diagnosed AL amyloidosis The study showed higher hematologic response rate in ITT population (53% vs 18%), patients …

Janssen’s Darzalex (daratumumab, SC) Combination Regimen Receives Health Canada’s Approval for Patients with Newly Diagnosed Light Chain Amyloidosis Read More »

Astellas and Seagen Report the US FDA’s Acceptance of Two sBLA for Padcev (enfortumab vedotin) in Locally Advanced or Metastatic Urothelial Cancer

Shots: The first sBLA is based on the P-III EV-301 trial assessing Padcev vs CT in ~ 600 patients with LA or mUC prior treated with PD-1/L1 inhibitor and platinum-based therapy. The first sBLA seeks to convert PADCEV’s accelerated approval to regular approval The second sBLA is based on cohort 2 of the P-II EV-201 …

Astellas and Seagen Report the US FDA’s Acceptance of Two sBLA for Padcev (enfortumab vedotin) in Locally Advanced or Metastatic Urothelial Cancer Read More »

Sanofi’s Sarclisa (isatuximab) Receives EC’s Approval for Adults with Relapsed and Refractory Multiple Myeloma

Shots: The approval is based on data from the P-III IKEMA study evaluating Sarclisa + carfilzomib and dexamethasone (Kd) vs Kd alone in 302 patients with relapsed MM across 69 centers spanning 16 countries Results: mPFS (not reached vs 19.15 mos.) at the time of the pre-planned interim analysis, reduction in the risk of disease …

Sanofi’s Sarclisa (isatuximab) Receives EC’s Approval for Adults with Relapsed and Refractory Multiple Myeloma Read More »

Roche’s Evrysdi (risdiplam) Receives Health Canada’s Approval for Spinal Muscular Atrophy in Adults and Children

Shots: The approval is based on two studies i.e. FIREFISH & SUNFISH. The FIREFISH study involves assessing Evrysdi in infants aged 2-7mos. with symptomatic Type 1 SMA, and the SUNFISH study assesses Evrysdi in children & young adults aged 2-25yrs. with Type 2 or 3 SMA Evrysdi demonstrated improvement in motor function, 29.3% of participants …

Roche’s Evrysdi (risdiplam) Receives Health Canada’s Approval for Spinal Muscular Atrophy in Adults and Children Read More »

BMS’ Opdivo (nivolumab) + Cabometyx (cabozantinib) Receive EC’s Approval for 1L Treatment of Advanced Renal Cell Carcinoma

Shots: The approval is based on P-III CheckMate -9ER trial involves assessing Opdivo + Cabometyx vs sunitinib in 651 patients with previously untreated advanced or metastatic RCC Results: @median follow-up of 10.6 mos.; m-PFS (16.6 vs 8.3mos.), ORR (55.7% vs 27.1%); CR (9.3% vs 4.3%); OS (40% reduction in risk of death), Grade 3+ AEs …

BMS’ Opdivo (nivolumab) + Cabometyx (cabozantinib) Receive EC’s Approval for 1L Treatment of Advanced Renal Cell Carcinoma Read More »

AstraZeneca’s Tagrisso (osimertinib) Receives the NMPA’s Approval for the Adjuvant Treatment of Patients with Early-Stage EGFR-Mutated Lung Cancer

Shots: The approval is based on results from the P-III ADAURA trial assessing Tagrisso (80mg, qd) vs PBO in 682 patients with stage IB, II, IIIA EGFRm NSCLC following complete tumor resection and adjuvant chemotherapy as indicated for three years or until disease recurrence Results: Improvement in DFS in all population, 83% reduction in the …

AstraZeneca’s Tagrisso (osimertinib) Receives the NMPA’s Approval for the Adjuvant Treatment of Patients with Early-Stage EGFR-Mutated Lung Cancer Read More »

Novartis’s Xolair (omalizumab) Prefilled Syringe for Self-Injection Receives the US FDA’s Approval Across All Indications

Shots: The US FDA has approved the sBLA for a new self-administration option for Xolair in patients across all approved indications in the US The use of the therapy across multiple indications is based on the efficacy and safety profile of Xolair in allergic asthma, CIU, and nasal polyps, supported with a clinical development program, …

Novartis’s Xolair (omalizumab) Prefilled Syringe for Self-Injection Receives the US FDA’s Approval Across All Indications Read More »

BMS Reports Results of Opdivo (nivolumab) + CT or Yervoy (ipilimumab) in P-III CheckMate -648 Trial for Unresectable Advanced or Metastatic ESCC

Shots: The P-III CheckMate -648 trial involves assessing Opdivo (nivolumab) + CT or Opdivo (3 mg/kg, q2w) + Yervoy (1 mg/kg, q6w up to 24mos.) in patients with unresectable advanced or metastatic ESCC Results: Both the combinations demonstrated OS benefits in PD-L1 positive and all-randomized populations Additionally, Opdivo + CT met its 1EP of PFS …

BMS Reports Results of Opdivo (nivolumab) + CT or Yervoy (ipilimumab) in P-III CheckMate -648 Trial for Unresectable Advanced or Metastatic ESCC Read More »

AbbVie Submits Regulatory Applications to the US FDA and EMA for Skyrizi (risankizumab) to Treat Psoriatic Arthritis

Shots: The submission is based on two P-III studies KEEPsAKE-1 & -2 studies involves assessing Skyrizi (150mg) vs PBO in adult patients with active PsA who had an inadequate response or intolerant to biologic therapy and/or non-biologic DMARDs respectively The 1EP and 2EP of both studies showed ACR20 response @24wks., improvements in disease activity, skin …

AbbVie Submits Regulatory Applications to the US FDA and EMA for Skyrizi (risankizumab) to Treat Psoriatic Arthritis Read More »

Biogen’s Tysabri (natalizumab, SC) Receives EC’s Marketing Authorization to Treat Relapsing-Remitting Multiple Sclerosis

Shots: The EC’s approval is based on data from DELIVER and REFINE studies evaluating the efficacy, PK, and PD of Tysabri (300mg, SC, q4w) vs Tysabri (300mg, IV, q4w) in patients with RRMS The results demonstrated that the Tysabri (SC) in both studies showed clinical benefits and well-characterized safety profile. The SC option expands the …

Biogen’s Tysabri (natalizumab, SC) Receives EC’s Marketing Authorization to Treat Relapsing-Remitting Multiple Sclerosis Read More »

The US FDA Extends Review Period of Olumiant’s sNDA to Treat Moderate to Severe Atopic Dermatitis

Shots: Lilly and Incyte reported that the US FDA has extended the review period of sNDA for baricitinib to treat adults with mod. to sev. atopic AD The FDA has extended the action date, allowing the time to review additional data analyses submitted by Lilly in response to recent information requests from the FDA. The …

The US FDA Extends Review Period of Olumiant’s sNDA to Treat Moderate to Severe Atopic Dermatitis Read More »

Passage Bio’s PBKR03 Receives EC’s Orphan Drug Designation for Krabbe Disease

Shots: The EC has granted ODD for PBKR03 for the treatment of Krabbe disease. The ODD was based on an EMA’s COMP positive opinion The designation provides potential benefits to the company, which include clinical protocol assistance, reduced regulatory fees, research grants, and up to 10 years of market exclusivity following regulatory approval The company …

Passage Bio’s PBKR03 Receives EC’s Orphan Drug Designation for Krabbe Disease Read More »

Exelixis Reports the US FDA’s Acceptance of IND for XB002 in Patients with Advanced Solid Tumors

Shots: The US FDA has accepted IND to assess the safety, tolerability, PK, and preliminary antitumor activity of XB002 in patients with advanced solid tumors. The P-I trial is expected to initiate in Q2’21 The preclinical data demonstrated that XB002 binds to tissue factors without affecting the coagulation cascade, in contrast with prior therapies in …

Exelixis Reports the US FDA’s Acceptance of IND for XB002 in Patients with Advanced Solid Tumors Read More »

Taiho Oncology’s Futibatinib (TAS-120) Receives the US FDA’s Breakthrough Therapy Designation for Advanced Cholangiocarcinoma

Shots: The designation is based on efficacy & safety results from the P-II FOENIX-CCA2 study involves assessing futibatinib (TAS-120) in patients with advanced cholangiocarcinoma. The findings will be presented at AACR 2021 The preliminary clinical evidence showed improvement on at least one clinical EPs over available therapy Futibatinib (TAS-120, PO) is a covalently-binding FGFR inhibitor …

Taiho Oncology’s Futibatinib (TAS-120) Receives the US FDA’s Breakthrough Therapy Designation for Advanced Cholangiocarcinoma Read More »

Lilly’s Taltz (ixekizumab) Receives Health Canada’s Approval for Pediatric Patients with Moderate to Severe Plaque Psoriasis

Shots: The approval is based on P-III study involves assessing Taltz (20mg for <25kg/40mg for 25-50kg/80 mg for >50 kg @12wks., with 40/80/160mg starting doses, respectively) vs PBO in 171 patients aged 6-<18 yrs. with mod. to sev. PsO Results: @ 12wks., patients achieving PASI 75 (89% vs 25%); patients achieving sPGA 0,1 (81% vs …

Lilly’s Taltz (ixekizumab) Receives Health Canada’s Approval for Pediatric Patients with Moderate to Severe Plaque Psoriasis Read More »

Kite Reports Submission of sBLA to the US FDA for Tecartus in Adult Patients with R/R Acute Lymphoblastic Leukemia

Shots: The sBLA is based on data from the P- I/II ZUMA-3 trial in adult patients age ≥18 years old for the treatment of adult patients with r/r B-cell ALL The focus of the study is to evaluate the safety and efficacy in adult patients with r/r ALL If approved, Tecartus will be the 1st …

Kite Reports Submission of sBLA to the US FDA for Tecartus in Adult Patients with R/R Acute Lymphoblastic Leukemia Read More »

BeyondSpring Submits NDA to the US FDA and China NMPA for Plinabulin to Prevent CT-Induced Neutropenia

Shots: The NDA submission is based on data from P-III PROTECTIVE-2 study evaluating Plinabulin + Neulasta vs Neulasta alone for the prevention of CIN The study met its 1EPs and 2EPs with an improvement in the rate of prevention of grade 4 neutropenia from 13.6% to 31.5%, the 2 EPs include DSN and ANC. The …

BeyondSpring Submits NDA to the US FDA and China NMPA for Plinabulin to Prevent CT-Induced Neutropenia Read More »

Sanofi’s Sarclisa (isatuximab-irfc) + Carfilzomib and Dexamethasone Receives the US FDA’s Approval for the Treatment of R/R Multiple Myeloma

Shots: The approval is based on P-III IKEMA study involves assessing of Sarclisa + carfilzomib and dexamethasone(kd) vs SOC in 302 patients with RRMM who had received 1 to 3 prior lines of treatment The result demonstrated a reduction in the risk of disease progression or death by 45%. At pre-planned interim analysis, median PFS …

Sanofi’s Sarclisa (isatuximab-irfc) + Carfilzomib and Dexamethasone Receives the US FDA’s Approval for the Treatment of R/R Multiple Myeloma Read More »

Novartis’ Kesimpta (ofatumumab) Receives the EC’s Approval for Adult Patients with Relapsing Multiple Sclerosis

Shots: The approval is based on P-III ASCLEPIOS I and II studies that involve assessing Kesimpta (20mg, monthly, SC) vs teriflunomide (14mg, PO, qd) in 1,882 adults aged 18-55yrs. with a confirmed diagnosis of RMS The study demonstrated a reduction of annual relapses by over 50% and achieved >30% relative risk reduction of 3mos. confirmed …

Novartis’ Kesimpta (ofatumumab) Receives the EC’s Approval for Adult Patients with Relapsing Multiple Sclerosis Read More »

Medtronic’s Harmony Transcatheter Pulmonary Valve Receives the US FDA’s Approval for Patients with Congenital Heart Disease

Shots: The approval is based on clinical data from the Harmony TPV clinical study that showed freedom from mortality & acceptable hemodynamic function @30days & 6mos. respectively The study demonstrated that patients treated with Harmony TPV experienced no significant reinterventions, reoperations or endocarditis @6mos. Harmony TPV is the first minimally invasive alternative for patients with …

Medtronic’s Harmony Transcatheter Pulmonary Valve Receives the US FDA’s Approval for Patients with Congenital Heart Disease Read More »

Roche and PTC’s Evrysdi (risdiplam) Receive the EC’s Approval as First and Only at Home Treatment For Spinal Muscular Atrophy

Shots: The approval is based on two studies i.e. FIREFISH and SUNFISH. The FIREFISH study involves assessing Evrysdi in infants aged 2-7mos. with symptomatic Type 1 SMA, and the SUNFISH study assesses Evrysdi in children & young adults with Type 2 or 3 SMA Evrysdi demonstrated a favourable efficacy & safety profile, with the safety …

Roche and PTC’s Evrysdi (risdiplam) Receive the EC’s Approval as First and Only at Home Treatment For Spinal Muscular Atrophy Read More »

Roche’s Tecentriq Receives CHMP’s Positive Opinion for 1L Treatment for Metastatic Non-Small Cell Lung Cancer

Shots: The CHMP’s recommendation is based on Phase III IMpower110 study assessing Tecentriq vs CT  in 572 PD-L1-selected, CT-naïve participants in a ratio (1:1) with stage IV non-squamous or squamous NSCLC Results: OS in people with high PD-L1 expression (7.1 vs 3.1mos.); safety profile is consistent with the current profile & no new safety signals …

Roche’s Tecentriq Receives CHMP’s Positive Opinion for 1L Treatment for Metastatic Non-Small Cell Lung Cancer Read More »

GSK and VIR Report EUA Submission to the US FDA for VIR-7831 to Treat COVID-19

Shots: The EUA submission is based on an interim analysis of the P-III COMET-ICE study assessing VIR-7831 vs PBO for the early treatment of COVID-19 in 583 adults at high risk of hospitalization The study demonstrated an 85% reduction in hospitalization. Due to evidence of profound efficacy, IDMC recommended stopping the trial for further enrollment …

GSK and VIR Report EUA Submission to the US FDA for VIR-7831 to Treat COVID-19 Read More »

Helius Medical’s PoNS Receives the US FDA’s Approval to Improve Gait in Multiple Sclerosis Patients

Shots: The approval is based on two clinical studies and a retrospective analysis of RWD. The device is intended to be used by prescription only as an adjunct to a supervised therapeutic exercise program in patients aged ⥸22yrs. The approval will help in improving the walking gait of people with MS in combination with a …

Helius Medical’s PoNS Receives the US FDA’s Approval to Improve Gait in Multiple Sclerosis Patients Read More »

GSK’s Benlysta (belimumab) Receives CHMP’s Opinion Recommending its Approval for Active Lupus Nephritis

Shots: EMA’s CHMP has adopted a positive opinion recommending the use of Benlysta (IV, SC) in combination with background immunosuppressive therapies for the treatment of adult patients with active LN The opinion is based on a P-III BLISS-LN study assessing Benlysta (IV,10 mg/kg) + standard therapy vs PBO in 448 adult patients with active LN …

GSK’s Benlysta (belimumab) Receives CHMP’s Opinion Recommending its Approval for Active Lupus Nephritis Read More »

BMS and bluebird bio’s Abecma (idecabtagene vicleucel) Receive the US FDA’s Approval as the First Anti-BCMA CAR T Cell Therapy for R/R Multiple Myeloma

Shots: The approval is based on the P-II  KarMMa trial that involves assessing Abecma in 127 patients with r/r MM prior treated with 3L+ therapy including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 Ab Results: ORR (72%); sCR (28%); mDoR (11mos.); responses were rapid and durable, with a median time to response of …

BMS and bluebird bio’s Abecma (idecabtagene vicleucel) Receive the US FDA’s Approval as the First Anti-BCMA CAR T Cell Therapy for R/R Multiple Myeloma Read More »

Astellas and Seagen Report EMA’s Acceptance of MAA for Enfortumab Vedotin to Treat Locally Advanced or Metastatic Urothelial Cancer

Shots: The MAA is based on the P-III EV-301 trial which involves assessing Enfortumab Vedotin vs CT in ~ 600 patients with LA/m-UC, prior treated with Pt.-based CT and a PD-1/L1 inhibitor The 1EPs of the trial is OS and 2EPs include PFS, ORR, DoR, DCR as well as the assessment of safety/tolerability and QoL …

Astellas and Seagen Report EMA’s Acceptance of MAA for Enfortumab Vedotin to Treat Locally Advanced or Metastatic Urothelial Cancer Read More »

Takeda Reports EMA’s Acceptance of TAK-003 for the Prevention of Dengue

Shots: The submission is based on a P-III TIDES trial assessing TAK-003 (0.5ml, SC) vs PBO in 20,000+ healthy children & adolescents aged 4-16yrs. to prevent dengue fever of any severity and due to any of the four dengue virus serotypes The trial met its 1EPs showed overall vaccine efficacy against VCD @12 mos. follow-up …

Takeda Reports EMA’s Acceptance of TAK-003 for the Prevention of Dengue Read More »

J&J Visions’ Acuvue Theravision with Ketotifen Receives MHLW’s Approval for Vision Correction and Allergic Eye Itch

Shots: The MHLW has approved Acuvue Theravision with Ketotifen as the first combination contact lens that provides vision correction and an antihistamine drug to relieve symptoms for people experiencing itchy allergy eyes Data demonstrated that 8 out of 10 contact lens wearers feel frustrated when their eye allergies interfere with normal contact lens wear. The …

J&J Visions’ Acuvue Theravision with Ketotifen Receives MHLW’s Approval for Vision Correction and Allergic Eye Itch Read More »

Chugai’s Polivy Receives MHLW’s Approval for Relapsed or Refractory Diffuse Large B-cell Lymphoma

Shots: The approval is based on results from P- Ib/II GO29365 study assessing Polivy (IV, 30 & 140mg) + BR vs BR alone in 80 DLBCL patients and P-II JO40762/P-DRIVE study that evaluated Polivy + BR therapy in 35 patients with R/R DLBCL not eligible for ASCT The P-II part of GO29365 study demonstrated CRR …

Chugai’s Polivy Receives MHLW’s Approval for Relapsed or Refractory Diffuse Large B-cell Lymphoma Read More »

Bayer’s Vitrakvi Receives MHLW’s Approval for the Treatment of NTRK Fusion-Positive Advanced or Recurrent Solid Tumors

Shots: The approval is based on the P-III NAVIGATE trial in adult and adolescent patients and P- I/II pediatric SCOUT trial involves assessing Vitrakvi for NTRK fusion-positive advanced or recurrent solid tumors The P-III NAVIGATE trial showed ORR (65.2%), CR (16.9%), and P-I/II SCOUT trial showed ORR (88.9%), CR (22.2%). In general, the studies demonstrated …

Bayer’s Vitrakvi Receives MHLW’s Approval for the Treatment of NTRK Fusion-Positive Advanced or Recurrent Solid Tumors Read More »

Incyte’s Pemazyre (pemigatinib) Receives MHLW’s Approval for Unresectable Biliary Tract Cancer with a FGFR2 Fusion Gene

Shots: The approval is based on the P-II FIGHT-202 study that involves assessing Pemazyre (13.5mg, qd, on a 21-day cycle) in adults aged ≥18yrs. with previously treated, LA/m- cholangiocarcinoma with documented FGFR2 fusion or rearrangement The study has three cohorts: Cohort A (FGFR2 fusions), Cohort B (other FGF/FGFR genetic alterations) or Cohort C (no FGF/FGFR …

Incyte’s Pemazyre (pemigatinib) Receives MHLW’s Approval for Unresectable Biliary Tract Cancer with a FGFR2 Fusion Gene Read More »

Zealand’s Zegalogue (dasiglucagon) Receives the US FDA’s Approval for the Treatment of Severe Hypoglycemia

Shots: The approval is based on efficacy results from P-III studies involve assessing Zegalogue vs PBO in children aged 6-17yrs. and in adults with T1D The study met its 1EPs showed an increase in blood glucose of ≥20 mg/dL within 45 minutes, the median time to blood glucose recovery (10min vs 30-45min), following injection of …

Zealand’s Zegalogue (dasiglucagon) Receives the US FDA’s Approval for the Treatment of Severe Hypoglycemia Read More »

Novo Nordisk Receives the US FDA’s Refusal to File Letter for Semaglutide to Treat T2D

Shots: The US FDA has issued refusal to file a letter covering the label expansion application for semaglutide (2.0mg/qw) for T2D, which was submitted on Jan 20, 2021 The US FDA has requested additional information including data relating to new manufacturing site Novo Nordisk plans to resubmit the application to FDA in Q2’21 Click here ­to­ …

Novo Nordisk Receives the US FDA’s Refusal to File Letter for Semaglutide to Treat T2D Read More »

Microsoft’s commitment to compliance in pharma and life sciences

Over a year has gone by since the beginning of the global pandemic, and it’s clear that the pharmaceutical industry and COVID-19 vaccines are at the center of everyone’s attention. Calls for quick and rigorous development of a COVID-19 vaccine have given way to the equally important demand for rapid distribution. Because of this unprecedented …

Microsoft’s commitment to compliance in pharma and life sciences Read More »

Kiniksa’s Arcalyst (rilonacept) Receives the US FDA’s Approval for the Treatment of Pericarditis

Shots: The approval is based on the P-III RHAPSODY trial involves assessing Arcalyst (SC, qw, 300mg followed by 160mg) vs PBO in adults and children aged ≥12yrs. in a ratio (1:1) with recurrent pericarditis The result showed a 96% reduction in the risk, with pain- free or minimal pain days (92% vs 40%), sustained reductions …

Kiniksa’s Arcalyst (rilonacept) Receives the US FDA’s Approval for the Treatment of Pericarditis Read More »

Kiniksa’s Arcalyst (rilonacept) Receives the US FDA’s Approval for the Treatment of Pericarditis

Shots: The approval is based on the P-III RHAPSODY trial involves assessing Arcalyst (SC, qw, 300mg followed by 160mg) vs PBO in adults and children aged ≥12yrs. in a ratio (1:1) with recurrent pericarditis The result showed a 96% reduction in the risk, with pain- free or minimal pain days (92% vs 40%), sustained reductions …

Kiniksa’s Arcalyst (rilonacept) Receives the US FDA’s Approval for the Treatment of Pericarditis Read More »

Merck’s Keytruda (pembrolizumab) Receives the EC’s Approval in Adults & Pediatric patients with R/R cHL

Shots: The approval is based on P-III KEYNOTE-204 study assessing Keytruda (200mg, IV, q3w) vs BV (1.8 mg/kg, IV, q3w) in 304 patients in a ratio (1:1) with r/r cHL who have failed ASCT or least two prior therapies when ASCT is not a treatment option Results: reduction in the risk of disease progression or …

Merck’s Keytruda (pembrolizumab) Receives the EC’s Approval in Adults & Pediatric patients with R/R cHL Read More »

Merck’s Keytruda (pembrolizumab) Receives the EC’s Approval in Adults & Pediatric patients with R/R cHL

Shots: The approval is based on P-III KEYNOTE-204 study assessing Keytruda (200mg, IV, q3w) vs BV (1.8 mg/kg, IV, q3w) in 304 patients in a ratio (1:1) with r/r cHL who have failed ASCT or least two prior therapies when ASCT is not a treatment option Results: reduction in the risk of disease progression or …

Merck’s Keytruda (pembrolizumab) Receives the EC’s Approval in Adults & Pediatric patients with R/R cHL Read More »

Merck Reports the US FDA’s Acceptance of NDA for Priority Review of Belzutifan (MK-6482) to Treat Patients with Von Hippel-Lindau Disease Associated RCC

Shots: The NDA is based on P-II study-004 trial involves assessing Belzutifan (120 mg, PO, qd) in 61 patients with VHL disease who had at least one measurable solid tumor localized to the kidney and did not require immediate surgery Results: It showed ORR of 36.1% while the 2EPs include DCR, DoR, time to response, …

Merck Reports the US FDA’s Acceptance of NDA for Priority Review of Belzutifan (MK-6482) to Treat Patients with Von Hippel-Lindau Disease Associated RCC Read More »

BioXcel’s BXCL501 Receives the US FDA’s Breakthrough Therapy Designation for Acute Treatment of Agitation Associated with Dementia

Shots: The BTD is based on a P-Ib/II TRANQUILITY study assessing BXCL501 in patients with acute treatment of agitation associated with dementia, including AD The results demonstrated significant reductions in agitation measures @2hrs. post-dose with both 30 and 60 mcg doses as measured by multiple scales. The dose dependent response has the potential to support …

BioXcel’s BXCL501 Receives the US FDA’s Breakthrough Therapy Designation for Acute Treatment of Agitation Associated with Dementia Read More »

Takeda Reports NDA Submission of Lanadelumab to the MHLW as a Preventive Treatment for Hereditary Angioedema Attack in Japan

Shots: The submission is based on P-III HELP study evaluating lanadelumab (300mg, q2w & q4w) vs PBO in 125 patients with HAE attack for 26wks. It also based on P-III HELP OLE study and interim results of P-III study that evaluated lanadelumab in Japanese patients. If approved, lanadelumab will be available as a pre-filled syringe …

Takeda Reports NDA Submission of Lanadelumab to the MHLW as a Preventive Treatment for Hereditary Angioedema Attack in Japan Read More »

Takeda Reports NDA Submission of Lanadelumab to the MHLW as a Preventive Treatment for Hereditary Angioedema Attack in Japan

Shots: The submission is based on P-III HELP study evaluating lanadelumab (300mg, q2w & q4w) vs PBO in 125 patients with HAE attack for 26wks. It also based on P-III HELP OLE study and interim results of P-III study that evaluated lanadelumab in Japanese patients. If approved, lanadelumab will be available as a pre-filled syringe …

Takeda Reports NDA Submission of Lanadelumab to the MHLW as a Preventive Treatment for Hereditary Angioedema Attack in Japan Read More »

Astellas Reports NDA Submission of Enfortumab Vedotin to the MHLW for Locally Advanced or Metastatic Urothelial Cancer in Japan

Shots: The submission is based on EV-301 and EV-201 trials in Japan. The P-III EV-301 trial assessing Enfortumab Vedotin vs CT in ~ 600 patients with LA/m-UC, prior treated with platinum-based CT and PD-1/L1 inhibitor The P-II EV-201 trial assessing Enfortumab Vedotin in ~128 patients with LA/m-UC, prior treated with PD-1/L1 inhibitor, including those who …

Astellas Reports NDA Submission of Enfortumab Vedotin to the MHLW for Locally Advanced or Metastatic Urothelial Cancer in Japan Read More »

Vertex’s VX-880 Receives the US FDA’s Fast Track Designation for T1D

Shots: The US FDA has granted FTD for VX-880 to treat T1D. The designation is designed to facilitate the development and expedite the review of treatments for serious condition and fill an unmet medical need Additionally, the company has initiated P-I/II trial for VX-880 in ~17 patients who have T1D with severe hypoglycemia and impaired …

Vertex’s VX-880 Receives the US FDA’s Fast Track Designation for T1D Read More »

Roche’s Ventana ALK (D5F3) CDx Assay Receives the US FDA’s Approval for the Treatment of ALK-Positive NSCLC

Shots: The US FDA has granted approval of Ventana ALK (D5F3) as a CDx assay to identify ALK+ NSCLC patients eligible for treatment with Pfizer’s drug Lorbrena (lorlatinib) The label expansion approval strengthens Roche’s commitment to personalized healthcare by providing lung cancer patients with access to more treatment options The Ventana ALK (D5F3) CDx assay …

Roche’s Ventana ALK (D5F3) CDx Assay Receives the US FDA’s Approval for the Treatment of ALK-Positive NSCLC Read More »

Kite’s Yescarta Receives the US FDA’s Approval for R/R Follicular Lymphoma

Shots: The approval is based on a P-II ZUMA-5 study assessing Yescarta (axicabtagene ciloleucel) in 146 patients aged ≥18rs. with r/r iNHL, prior treated with at least 2L of systemic therapy The P-II ZUMA-5 study showed 91% of patients responded to a single infusion of Yescarta, including ~74% of patients in a continued remission @18mos.; …

Kite’s Yescarta Receives the US FDA’s Approval for R/R Follicular Lymphoma Read More »

Roche to Withdraw Tecentriq Indication in Metastatic Bladder Cancer in the US

Shots: The company reported the voluntary withdrawal of the Tecentriq (atezolizumab) indication in the US for prior-platinum treated mUC, bladder cancer The withdrawal was made in consultation with the US FDA to review the accelerated approvals with confirmatory trials that have not met 1EPs and have yet to gain regular approvals. The decision does not …

Roche to Withdraw Tecentriq Indication in Metastatic Bladder Cancer in the US Read More »

Takeda Reports NDA Submission to Import and Distribute Moderna’s mRNA-1273 in Japan

Shots: Takeda has submitted an NDA to MHLW to import and distribute Moderna’s (mRNA-1273/ TAK-919) in Japan Takeda is conducting a P- I/II study assessing the safety and immunogenicity of two vaccinations of TAK-919 (100μg, given 28 days apart) in 200 participants aged ≥20yrs. followed through 12mos. after the second vaccination. Takeda has completed enrollment …

Takeda Reports NDA Submission to Import and Distribute Moderna’s mRNA-1273 in Japan Read More »

Roche’s Actemra/RoActemra (tocilizumab) Receives the US FDA’s Approval as the First Biologic for Systemic Sclerosis-Associated Interstitial Lung Disease

Shots: The approval is based on P-III focuSSced trial assessing Actemra (SC, 162mg) vs PBO in a ratio (1:1) in 212 adults with SSc. The data from P-I/II fascinate study also supported the approval The P-III study did not meet its 1EPs i.e. change from baseline @48wks. in mRSS. In subgroup patients, decline in mean …

Roche’s Actemra/RoActemra (tocilizumab) Receives the US FDA’s Approval as the First Biologic for Systemic Sclerosis-Associated Interstitial Lung Disease Read More »

Regeneron and Sanofi Report the US FDA’s Acceptance of sBLA for Review of Dupixent to Treat Moderate-to-Severe Asthma in Children

Shots: The sBLA is based on P-III study assessing Dupixent + SOC maintenance therapy in children aged 6-11yrs. with moderate-to-severe asthma with type 2 inflammation The study resulted in a reduction of severe asthma attacks and rapidly improved lung function within 2wks, safety results were generally consistent with the known safety profile of Dupixent. Dupixent …

Regeneron and Sanofi Report the US FDA’s Acceptance of sBLA for Review of Dupixent to Treat Moderate-to-Severe Asthma in Children Read More »

Pfizer’s Lorbrena (lorlatinib) Receives FDA’s Approval for Expanding its Indication to Treat ALK-Positive Metastatic Lung Cancer

Shots: The approval is based on P-III CROWN study assessing Lorbrena vs Xalkori (crizotinib) monthx in 296 people in a ratio (1:1) with prior untreated advanced ALK-positive NSCLC The P-III study resulted in a 72% reduction in risk of progression or death as assessed by BICR. The prespecified exploratory analysis showed IC-ORR was (82% vs …

Pfizer’s Lorbrena (lorlatinib) Receives FDA’s Approval for Expanding its Indication to Treat ALK-Positive Metastatic Lung Cancer Read More »

BeiGene’s Brukinsa (zanubrutinib) Receives Health Canada’s Approval for Waldenström’s Macroglobulinemia

Shots: The approval is based on a P-III APSEN study involve assessing Brukinsa vs ibrutinib in 201 patients with r/r or TN WM who harbor an MYD88 mutation (MYD88MUT). The recommended dose of the therapy is 320mg and is expected to be available in Canada imminently The P-III APSEN study demonstrated clinical benefit with advantages …

BeiGene’s Brukinsa (zanubrutinib) Receives Health Canada’s Approval for Waldenström’s Macroglobulinemia Read More »

Merck Reports the US FDA’s Acceptance of NDA for Review of Gefapixant to Treat Refractory or Unexplained Chronic Cough

Shots: The NDA submission is based on two P-III studies (COUGH-1 & -2) involves assessing gefapixant (45/15mg, bid) vs PBO in 2,044 patients with RCC & UCC In both the studies, gefapixant resulted in the reduction of in 24hrs.-cough frequency @12 & 24wks respectively. Data were presented at ERS 2020 Gefapixant is a selective P2X3 …

Merck Reports the US FDA’s Acceptance of NDA for Review of Gefapixant to Treat Refractory or Unexplained Chronic Cough Read More »

BMS’ Opdivo (nivolumab) + Cabometyx (cabozantinib) Receive CHMP’s Positive Opinion as 1L Treatment for Advanced Renal Cell Carcinoma

Shots: The CHMP recommendation is based on P-III CheckMate -9ER trial involves assessing Opdivo + Cabometyx vs sunitinib in 651 patients with previously untreated advanced or metastatic RCC The P-III result showed improvements in PFS, OS and ORR with consistent efficacy benefits observed across key subgroups of patients and was well tolerated, with a low …

BMS’ Opdivo (nivolumab) + Cabometyx (cabozantinib) Receive CHMP’s Positive Opinion as 1L Treatment for Advanced Renal Cell Carcinoma Read More »

Regeneron’s Ab Cocktail (casirivimab + imdevimab) Receives the CHMP’s Positive Opinion to Treat COVID-19

Shots: The EMA’s CHMP has recommended the use of casirivimab + imdevimab in patients with confirmed COVID-19 who do not require oxygen supplementation and who are at high risk of progressing to severe COVID-19 The CHMP scientific opinion supports national decision making within EU states on the use of the antibodies, prior to the future …

Regeneron’s Ab Cocktail (casirivimab + imdevimab) Receives the CHMP’s Positive Opinion to Treat COVID-19 Read More »

Johnson & Johnson Receives the US FDA’s EUA for COVID-19 Vaccine

Shots: The FDA has issued a EUA for a single-shot COVID-19 vaccine to prevent COVID-19 in individuals aged ≥18yrs. The company plans to file BLA to the US FDA in 2021 The EUA is based on a P-III ENSEMBLE study that demonstrated 85% efficacy in preventing severe disease across all regions and protect against COVID-19 …

Johnson & Johnson Receives the US FDA’s EUA for COVID-19 Vaccine Read More »

BridgeBio and Origin’s Nulibry (fosdenopterin) Receives the US FDA’s Approval as the First Therapy to Reduce the Risk of Mortality in Patients with MoCD Type A

Shots: The FDA has approved Nulibry based on three clinical trials compared to data from a natural history study in patients with MoCD Type A The results from three clinical trials showed improvement in overall survival and an 82% reduction in the risk of death, @3yrs. probability of survival in rcPMP-treated patients (84% vs 55%), …

BridgeBio and Origin’s Nulibry (fosdenopterin) Receives the US FDA’s Approval as the First Therapy to Reduce the Risk of Mortality in Patients with MoCD Type A Read More »

Regeneron Reports IDMC Clearance of Efficacy for REGEN-COV (Casirivimab + Imdevimab) in P-III COVID-19 Outpatient Outcomes Trial

Shots: Regeneron reported changes to the P-III study assessing REGEN-COV in non-hospitalized patients with COVID-19, following recommendations from the IDMC The IDMC found clear clinical efficacy in reducing the rate of hospitalization and death with both doses (1,200 & 2,400 mg) of REGEN-COV vs PBO. The company will immediately stop enrolling patients in the PBO …

Regeneron Reports IDMC Clearance of Efficacy for REGEN-COV (Casirivimab + Imdevimab) in P-III COVID-19 Outpatient Outcomes Trial Read More »

AbbVie’s Humira (adalimumab) Receives the US FDA’s Approval to Treat Pediatric Patients with Ulcerative Colitis

Shots: The approval is based on P-III ENVISION I study that involves assessing Humira (SC) vs PBO in pediatric patients aged 4-17yrs. with mod. to sev. UC The study demonstrated that 60% of patients taking the higher dosage achieved clinical remission per PMS @8wks induction period and 45% of patients who responded @8wks, achieved remission …

AbbVie’s Humira (adalimumab) Receives the US FDA’s Approval to Treat Pediatric Patients with Ulcerative Colitis Read More »

Pfizer Reports the US FDA’s Acceptance and Priority Review of BLA for Ticovac (Tick-Borne Encephalitis Vaccine)

Shots: The US FDA has accepted the PR of BLA for TicoVac to prevent TBE in patients aged ≥1year with an anticipated PDUFA date in Aug 2021. If approved, the vaccine may help reduce the risk of TBE for people traveling to endemic areas The BLA is based on clinical trials that assess TicoVac across …

Pfizer Reports the US FDA’s Acceptance and Priority Review of BLA for Ticovac (Tick-Borne Encephalitis Vaccine) Read More »