Priority Review

Boehringer Ingelheim and Eli Lilly Report the US FDA’s Acceptance of sNDA and Granted Priority Review for Jardiance (empagliflozin) to Treat HFpEF

Shots: The sNDA is based on P-III EMPEROR-Preserved trial evaluates the safety and efficacy of Jardiance (10mg, qd) vs PBO in 5,988 patients with chronic HFpEF with/out diabetes The results showed a 21% vs 40% reduction in relative risk for the composite 1EPs of CV death or hospitalization in adults with HF with LVEF The …

Boehringer Ingelheim and Eli Lilly Report the US FDA’s Acceptance of sNDA and Granted Priority Review for Jardiance (empagliflozin) to Treat HFpEF Read More »

Novartis Reports the US FDA and EMA’s Acceptance of sBLA for Kymriah (tisagenlecleucel) and Granted Priority Review to Treat R/R Follicular Lymphoma

Shots: The submissions are based on the P-II ELARA trial evaluates the efficacy and safety of Kymriah in adult patients with r/r FL after at least two prior therapies over 30 sites in 12 countries globally The trial met its 1EPs i.e., the therapy showed a robust response in heavily pretreated patients & the safety …

Novartis Reports the US FDA and EMA’s Acceptance of sBLA for Kymriah (tisagenlecleucel) and Granted Priority Review to Treat R/R Follicular Lymphoma Read More »

Regeneron Reports the US FDA’s Acceptance of Priority Review for REGEN-COV for the Treatment and Prophylaxis of COVID-19

Shots: The US FDA has accepted for priority review a BLA for REGEN-COV (casirivimab and imdevimab) to treat COVID-19 in non-hospitalized patients and as prophylaxis in certain individuals The BLA is based on P-III study assessing efficacy & safety of REGEN-COV to treat non-hospitalized patients already infected with SARS-CoV-2, and to prevent symptomatic infection in …

Regeneron Reports the US FDA’s Acceptance of Priority Review for REGEN-COV for the Treatment and Prophylaxis of COVID-19 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 »

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 »

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 »

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 »

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 »

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 »

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 »

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 »

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 »

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 »

Incyte Reports the US FDA’s Acceptance of sNDA for Priority Review of Jakafi (ruxolitinib) to Treat Chronic Graft Versus Host Disease

Shots: The sNDA submission is based on the P-III REACH3 study assessing ruxolitinib vs best available therapy in adult and pediatric patients ≥12yrs. with steroid-refractory chronic GVHD. The findings were recently presented at the 62nd ASH Annual Meeting & Exposition The study showed greater ORR @24wks. i.e 1EPs (49.7% vs 25.6%); @24wks. mFFS (not reached …

Incyte Reports the US FDA’s Acceptance of sNDA for Priority Review of Jakafi (ruxolitinib) to Treat Chronic Graft Versus Host Disease Read More »

Incyte Reports the US FDA’s Acceptance of NDA for Priority Review of Ruxolitinib Cream to Treat Atopic Dermatitis

Shots: The NDA is based on two P-III TRuE-AD 1 & 2 assessing ruxolitinib cream (0.75%/1.5%, bid) vs vehicle (non-medicated cream) in 600 patients aged ≥12yrs. in a ratio (2:2:1) diagnosed with AD for at least 2yrs. and who were candidates for topical therapy Patients who completed an assessment @8wks. were offered participation in the …

Incyte Reports the US FDA’s Acceptance of NDA for Priority Review of Ruxolitinib Cream to Treat Atopic Dermatitis Read More »

Incyte makes bid to join the checkpoint inhibitor club

Incyte has filed its PD-1 inhibitor retifanlimab with the FDA, seeking approval to treat a form of anal cancer associated with human papillomavirus (HPV) and HIV infections. The application covers the use of the intravenous PD-1 inhibitor for adults with advanced squamous cell carcinoma of the anal canal (SCAC) who have progressed on, or who …

Incyte makes bid to join the checkpoint inhibitor club Read More »

Incyte Reports Acceptance and Priority Review of BLA for Retifanlimab to Treat Patients with Squamous Cell Carcinoma of the Anal Canal (SCAC)

Shots: The BLA submission is based on P-II POD1UM-202 trial involves assessing of retifanlimab (500 mg, q4w) in 94 patients with locally advanced or metastatic SCAC who have progressed on or are intolerant of standard platinum-based CT The study demonstrates 14% ORR for retifanlimab monotherapy as determined by ICR using RECIST v1.1 and mDOR of …

Incyte Reports Acceptance and Priority Review of BLA for Retifanlimab to Treat Patients with Squamous Cell Carcinoma of the Anal Canal (SCAC) Read More »

Roche Reports the US FDA’s Acceptance of sNDA and Granted Priority Review for Esbriet (pirfenidone) to Treat UILD

Shots: The sNDA submission is based on a P-II study that involves assessing Esbriet vs PBO in patients aged ≥18-85yrs. with progressive fibrosing UILD for 24wks. The anticipated PDUFA date is May’2021 Results: Over 24wks. predicted median change in FVC measured by home spirometry (-87.7 vs -157.1 mL); change in percent predicted DLco and 6MWD …

Roche Reports the US FDA’s Acceptance of sNDA and Granted Priority Review for Esbriet (pirfenidone) to Treat UILD Read More »

Omeros Reports the US FDA’s Acceptance and Priority Review of BLA for Narsoplimab (OMS721) to Treat HSCT-TMA

Shots: The US FDA has accepted the BLA and granted the PR with an anticipated PDUFA date as Jul 17, 2021 The BLA filing marks the milestone on the path to commercialization of narsoplimab Narsoplimab is an investigational mAb targeting MASP-2 has received the US FDA’s BTD and ODD to the FDA for each of …

Omeros Reports the US FDA’s Acceptance and Priority Review of BLA for Narsoplimab (OMS721) to Treat HSCT-TMA Read More »

Astellas Reports the US FDA’s Acceptance of Priority Review for its NDO Therapy

Shots: The US FDA has accepted the PR for NDA of mirabegron (oral suspension) and sNDA for Myrbetriq (mirabegron, tablets) for neurogenic detrusor overactivity (NDO) in pediatric patients aged ≥ 3yrs. with anticipated PDUFA date as Mar 28, 2021 The NDA & sNDA is based on P-III study assessing efficacy, safety, tolerability and PK of …

Astellas Reports the US FDA’s Acceptance of Priority Review for its NDO Therapy Read More »

Provention Bio Reports Submission of BLA and Priority Review to the US FDA for Teplizumab to Prevent T1D

Shots: The BLA for teplizumab to delay or prevention of clinical T1D in at-risk individuals has been filed to the US FDA. The FDA has also granted Provention’s request for Priority Review and assigned anticipated PDUFA date as Jul 02, 2021 The company is actively working with the FDA to support their review while planning …

Provention Bio Reports Submission of BLA and Priority Review to the US FDA for Teplizumab to Prevent T1D Read More »

Pfizer Report the US FDA’s Acceptance and Priority Review of sNDA for Lorbrena (lorlatinib) to treat (ALK)-Positive (NSCLC)

Shots: The US FDA has accepted for Priority Review the sNDA is based on the pivotal CROWN study and is being reviewed by the FDA under its Real-Time Oncology Review (RTOR) CROWN is P-lll, parallel 2-arm trial in which 296 previously untreated advanced ALK-positive NSCLC were randomized in ratio 1:1 to receive LORBRENA monotherapy (n=149) …

Pfizer Report the US FDA’s Acceptance and Priority Review of sNDA for Lorbrena (lorlatinib) to treat (ALK)-Positive (NSCLC) Read More »

Sanofi’s Avalglucosidase alfa Receives US FDA’s Priority Review as Enzyme Replacement Therapy for Pompe Disease

Shots: The US FDA accepted for priority review the BLA of avalglucosidase alfa for long-term enzyme replacement therapy for the treatment of patients with Pompe disease. The target action date for the FDA decision is May 18, 2021 The BLA is based on positive data from two trials: P-III COMET study and P-II mini-COMET study …

Sanofi’s Avalglucosidase alfa Receives US FDA’s Priority Review as Enzyme Replacement Therapy for Pompe Disease Read More »

G1 Therapeutics Reports the US FDA Acceptance and Priority Review of NDA for Trilaciclib to Treat SCLC

Shots: The NDA submission is based on three clinical studies in which Trilaciclib was administered prior to chemotherapy treatment in patients with SCLC and has demonstrated robust myelopreservation benefits. The company anticipates the PDUFA date as Feb 15, 2021 G1 is making Trilaciclib available to SCLC patients in the US, who are unable to enter …

G1 Therapeutics Reports the US FDA Acceptance and Priority Review of NDA for Trilaciclib to Treat SCLC Read More »

AstraZeneca’s Imfinzi (durvalumab) Receives the US FDA’s Priority Review for Less Frequent Fixed-Dose Use in NSCLC and Bladder Cancer

Shots: The US FDA has accepted sBLA and granted PR to Imfinzi (1500mg) for a new 4wks. FD regimen to treat patients with unresectable Stage III NSCLC after CT and prior treated advanced bladder cancer, consistent with the approved dosing in ES-SCLC The company anticipates the PDUFA date in Q4’20. The sBLA is based on …

AstraZeneca’s Imfinzi (durvalumab) Receives the US FDA’s Priority Review for Less Frequent Fixed-Dose Use in NSCLC and Bladder Cancer Read More »

Regeneron Reports the US FDA’s Acceptance of Evinacumab’s BLA for Priority Review as a Treatment for Patients with HoFH

Shots: The BLA is based on P-III study evaluating the efficacy and safety of evinacumab (15 mg/kg, IV, q4w) in 65 patients aged ≥12yrs. with HoFH. The 1EPs of the study is reduction of LDL-C from baseline The expected PDUFA date for the therapy as Feb 11, 2021. The US FDA has granted BT designation …

Regeneron Reports the US FDA’s Acceptance of Evinacumab’s BLA for Priority Review as a Treatment for Patients with HoFH Read More »

Merck and Bayer Receive the US FDA’s Priority Review for Vericiguat to Reduce the Risk of CV Death in Patients with Symptomatic Chronic Heart Failure with HFrEF

Shots: The NDA is based on P-III VICTORIA study assessing vericiguat (qd, (titrated up to 10mg) vs PBO when given in combination with available HF therapies in ~5,050 patients with worsening CHF, reduced left VEF of <45% within 12mos. prior to randomization following a decompensation event The 1EPs is the composite of time to the …

Merck and Bayer Receive the US FDA’s Priority Review for Vericiguat to Reduce the Risk of CV Death in Patients with Symptomatic Chronic Heart Failure with HFrEF Read More »