Alnylam

Alnylam gets NICE backing for porphyria therapy Givlaari

UK cost-effectiveness watchdog NICE is set to recommend NHS use of Alnylam’s gene-silencing therapy Givlaari in England and Wales for the rare disease acute hepatic porphyria (AHP), after its advisors issued a positive verdict on the drug. Givlaari (givosiran) has been recommended as a treatment option for people aged 12 and over with AHP, a …

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Alnylam launches digital tool for AATR amyloidosis patients

Alnylam has been a pioneer in hereditary ATTR amyloidosis, getting approval in 2018 for the first drug to treat the rare disease, and has now launched a digital companion to help patients track their symptoms. The new app – called STAR – allows patients to record their symptoms and share them with healthcare professionals to …

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Exclusive Interview with PharmaShots: Ron Elwell of IPM.ai Shares Insight on the Simplification of Patient Recruitment by Applying AI and ML to Real-World Data

In an interview with PharmaShots, Ron Elwell, President, and Founder of Swoop and IPM.ai shared his views on the Real Chemistry Clinical Trial Recruitment System that is designed to accelerate clinical investigations and lead to faster patient outcomes with less risk. Shots: Real Chemistry’s IPM.ai accelerates clinical investigation & commercialization of precision medicines leveraging AI …

Exclusive Interview with PharmaShots: Ron Elwell of IPM.ai Shares Insight on the Simplification of Patient Recruitment by Applying AI and ML to Real-World Data Read More »

PharmaShots Weekly Snapshots (Aug 09 – 13, 2021)

Junshi and Coherus’s Toripalimab Receive the US FDA’s Breakthrough Therapy Designation as 1L Treatment of Nasopharyngeal Carcinoma Published: Aug 13, 2021 | Tags: Junshi, Coherus, Toripalimab, US, FDA, Breakthrough Therapy Designation, Nasopharyngeal Carcinoma Celltrion’s Regdanvimab (CT-P59) Receives the ANVISA’s EUA for the Treatment of COVID-19 in Brazil Published: Aug 13, 2021 | Tags: Celltrion, Regdanvimab, …

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Alnylam Reports Completion of Patient Enrollment in P-III HELIOS-B Study for Vutrisiran to Treat ATTR Amyloidosis with Cardiomyopathy

Shots: The company has completed the enrolment in the P-III HELIOS-B study evaluates the efficacy and safety of vutrisiran (25 mg, SC, q3M, for ~ 36 mos.) vs PBO in 600 patients in a ratio (1:1) with ATTR amyloidosis with cardiomyopathy across 123 activated sites in 32 countries The 1EP of the study is to …

Alnylam Reports Completion of Patient Enrollment in P-III HELIOS-B Study for Vutrisiran to Treat ATTR Amyloidosis with Cardiomyopathy Read More »

PharmaShots Weekly Snapshots (July 26 – 30, 2021)

Roche Publishes the Results of Evrysdi (risdiplam) in FIREFISH Study to Treat of Type 1 SMA at NEJM Published: July 30, 2021 | Tags: Roche, Evrysdi, risdiplam, FIREFISH Study, Type 1 SMA, NEJM BeiGene Reports Results of Brukinsa (zanubrutinib) in P-III SEQUOIA Trial for the Treatment of Chronic Lymphocytic Leukemia Published: July 30, 2021 | …

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Novartis tries to rescue stalled inclisiran filing with FDA

Novartis has tried to get its marketing application for high cholesterol therapy inclisiran in the US back on track, after the FDA rejected it last year, by changing the factory that makes the drug. The US regulator issued Novartis with a complete response letter (CRL) for inclisiran in December, citing “unresolved facility inspection-related conditions” at …

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PharmaShots Weekly Snapshots (May 31 – June 4, 2021)

Tonix Pharmaceuticals Presents Results of TNX-102 SL in P-III RELIEF study for the Management of Fibromyalgia at ASCP 2021 Published: June 4, 2021 | Tags: Tonix Pharmaceuticals, TNX-102 SL, P-III, RELIEF Study, Fibromyalgia, ASCP 2021 Dermavant Submits NDA to the US FDA for Tapinarof Cream to Treat Plaque Psoriasis Published: June 4, 2021 | Tags: …

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Alnylam Reports Completion of Patient Enrollment in P-III APOLLO-B Study for Patisiran to Treat Transthyretin-Mediated (ATTR) Amyloidosis with Cardiomyopathy

Shots: The company has completed the enrolment in the P-III APOLLO-B study to evaluate the efficacy & safety of patisiran vs PBO in a ratio (2:1) in 300 patients with ATTR amyloidosis with cardiomyopathy across 90 sites in 20+ countries. The results are expected in mid-2022 The 1EPs of the trial is the change from …

Alnylam Reports Completion of Patient Enrollment in P-III APOLLO-B Study for Patisiran to Treat Transthyretin-Mediated (ATTR) Amyloidosis with Cardiomyopathy Read More »

Sanofi eyes 2022 filing for haemophilia drug fitusiran, despite safety issue

Sanofi’s much anticipated drug for both haemophilia A and B – fitusiran – could be filed in 2022 despite a problem with blood clots in some patients in late-stage testing.  In its quarterly results update, Sanofi notes that a revised dosing schedule is being tested in its phase 3 programme to try to reduce the …

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PharmaShots Weekly Snapshots (Apr 19 – 23, 2021)

BMS’ Opdivo + Yervoy and Onureg Receive EC’s CHMP Positive Opinion for MPM and AML Published: Apr 23, 2021 | Tags: BMS, Opdivo, Yervoy, Onureg, Receive, EC, CHMP, Positive Opinion, MPM, AML Gilead Reports NDA Submission of Filgotinib to PMDA for Ulcerative Colitis in Japan Published: Apr 23, 2021 | Tags: Gilead, Reports, NDA, Submission, …

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Alnylam Presents Results of Vutrisiran in P-III HELIOS-A Study for Patients with hATTR Amyloidosis with Polyneuropathy at AAN 2021

Shots: The P-III HELIOS-A study assessing Vutrisiran (25mg, SC, once every 3mos.) vs patisiran (0.3 mg/kg, IV, q3w) in 164 patients in a ratio (3:1) with hATTR amyloidosis with polyneuropathy The study met its 1EPs & 2EPs i.e. change from baseline in mNIS+7 @9mos. & improvement in QoL assessed by Norfolk QoL-DN and gait speed …

Alnylam Presents Results of Vutrisiran in P-III HELIOS-A Study for Patients with hATTR Amyloidosis with Polyneuropathy at AAN 2021 Read More »

PharmaShots Weekly Snapshots (Mar 29 – Apr 02, 2021)

Pfizer and BioNTech Report Results of BNT162b2 to Prevent COVID-19 Published: Apr 2, 2021 | Tags: Pfizer and BioNTech, Report, Results, BNT162b2, Prevent, COVID-19 Acceleron Presents Results of Sotatercept in P-II PULSAR Trial for the Treatment of Pulmonary Arterial Hypertension, Published in NEJM Published: Apr 2, 2021 | Tags: Acceleron, Presents, Results, Sotatercept, P-II PULSAR …

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Alnylam Report Results of Lumasiran in P-III ILLUMINATE-A Study for Primary Hyperoxaluria Type 1, Published in NEJM

Shots: The P-III ILLUMINATE-A Study involves assessing Lumasiran vs PBO in 39 patients with age 6 or older in a ratio (2:1) for PH1 The study met its 1EPs demonstrated a 53.5% reduction in urinary oxalate excretion from baseline to 6 mos. i.e. change in 24 hrs. The 2EPs showed 84% of patients achieved normal …

Alnylam Report Results of Lumasiran in P-III ILLUMINATE-A Study for Primary Hyperoxaluria Type 1, Published in NEJM Read More »

PharmaShots Weekly Snapshot (Jan 4-8, 2020)

Almirall and Tyris Collaborate to Develop Next Generation Gene Therapies Published: Jan 7, 2020 | Tags: Almirall, Tyris, Collaborate, Develop, Next Generation, Gene Therapies Sarepta Reports Mixed Results of SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) in Part 1 of Study 102 for DMD    Published: Jan 7, 2020 | Tags: Sarepta, Reports, Mixed Results, SRP-9001, (rAAVrh74.MHCK7.micro-dystrophin), Part 1, Study 102, …

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Alnylam Reports Results of Vutrisiran in P-III HELIOS-A Study for Patients with hATTR Amyloidosis with Polyneuropathy

Shots: The P-III HELIOS-A study assessing vutrisiran (25mg, SC, once every 3mos.) vs patisiran (0.3 mg/kg, IV, q3w) in 164 patients in a ratio (3:1) with hATTR amyloidosis with polyneuropathy The study met its 1EPs & 2EPs i.e. change from baseline in the mNIS+7 @9mos. & changes in QoL assessed by Norfolk QoL-DN and gait …

Alnylam Reports Results of Vutrisiran in P-III HELIOS-A Study for Patients with hATTR Amyloidosis with Polyneuropathy Read More »

FDA rejects Novartis’ cholesterol drug after factory inspection issue

Novartis’ cholesterol drug inclisiran has hit a speed bump with the FDA, which has rejected the potential blockbuster because a manufacturing facility has yet to be inspected. Matters appear to have been complicated by the pandemic and the travel disruption that it has caused, which is preventing an FDA team from visiting the plant in …

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Insights+: The US FDA New Drug Approvals in November 2020

The US FDA has approved 5 NDAs and 1 BLA in Nov 2020, leading to treatments for patients and advances in the health care industry. The Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) have approved 96 novel products so far in 2020, including 6 in Nov 2020. …

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PharmaShots Weekly Snapshot (Nov 23 – 27, 2020)

Merck Collaborates with Siemens for the Digitalization of Production Published: Nov 26, 2020 | Tags: Agreement, Collaborative, Digitalization, Merck, Siemens, Sign CoSara Receives CDSCO’s Approval for its Saragene COVID-19 2-Gene Multiplex Test Published: Nov 26, 2020 | Tags: approval, CDSCO, Co-Diagnostics JV CoSara, COVID-19 2-gene multiplex RT-PCR, India, Manufacture, receives, Saragene, Sell, Test PostEra Collaborates …

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Alnylam’s Oxlumo (lumasiran) Receives the US FDA’s Approval for Treatment of Primary Hyperoxaluria Type 1 in Pediatric and Adult Patients

Shots: The approval is based on P-III ILLUMINATE-A & -B trials. The studies demonstrating reductions in urinary oxalate and encourage safety and tolerability in pediatric and adult patients The ILLUMINATE-A showed that Oxlumo met its 1EP i.e. change in 24hrs. (65% vs 12%) compared to PBO, the study also achieved significant results for all 6 …

Alnylam’s Oxlumo (lumasiran) Receives the US FDA’s Approval for Treatment of Primary Hyperoxaluria Type 1 in Pediatric and Adult Patients Read More »

FDA approves Alnylam’s ultra-rare disease drug Oxlumo

The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. Oxlumo (lumasiran) was also approved in the European Union last week, making it the third from Alnylam’s pipeline of RNA interference therapeutics to make it to market. The approval will mean …

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Sanofi/Alnylan’s haemophilia drug hits another safety speed-bump

Sanofi’s attempt to take on Roche with a ground-breaking approach to treating haemophilia has hit a setback after the company voluntarily placed its late-stage trials of fitusiran on hold because of safety issues. The news that phase 3 development has been put on hold was not announced by the French pharma, but in a joint …

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PharmaShots Weekly Snapshot (Sept 28 – Oct 1, 2020)

Sobi & Selecta Report Results of SEL-212 in P-II COMPARE Study for Chronic Refractory Gout Published: Oct 1, 2020 | Tags: Chronic Refractory Gout, COMPARE Study, Important Clinical Improvement, P-II, patients, reports, results, SEL-212, Selecta Biosciences, Sobi 2. Moderna’s mRNA-1273 COVID-19 Vaccine Demonstrate Immune Responses in Older Adults Published: Oct 1, 2020 | Tags: COVID-19, …

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Alnylam Reports Results of Lumasiran in P-III ILLUMINATE-B Study for Primary Hyperoxaluria Type 1 in Children Under the Age of Six

Shots: The P-III ILLUMINATE-B study involves assessing Lumasiran in 18 patients with PH1 under the age of 6 (3-72mos.), with an eGFR >45 mL/min/1.73 m2 or normal serum creatinine if less than 12mos. old at 9 study sites in 5 countries across the globe The study demonstrated a reduction in the hepatic production of oxalate across all …

Alnylam Reports Results of Lumasiran in P-III ILLUMINATE-B Study for Primary Hyperoxaluria Type 1 in Children Under the Age of Six Read More »

Alnylam, gene-silencing and biotech in 2020: the pharmaphorum podcast

Alnylam’s Brendan Martin joined the pharmaphorum podcast for episode 23 to talk about his company’s work in gene-silencing and how it could offer a route to target the current coronavirus pandemic. Brendan joined Alnylam in 2016 as one of the company’s first Europe-based employees and has helped to build and establish a presence in the …

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Alnylam’s Anant Murthy on making it as a US biotech in Europe

Alnylam’s European head of market access and GM of mid-size markets, Anant Murthy, shares his perspective on how a US biotech can adapt to the many unique challenges of the EU market. Though they represent the two biggest markets for pharma companies, the EU and US have some stark differences that can often catch out …

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UK patients get early access to Alnylam’s ultra-rare disease drug lumasiran

The UK’s regulator has granted early access to Alnylam’s ultra-rare disease drug lumasiran, allowing patients with primary hyperoxaluria type 1 to gain access ahead of a regulatory decision by the European Commission.  The Medicines and Healthcare products Regulatory Agency (MHRA) gave a positive opinion for lumasiran through its Early Access to Medicines Scheme (EAMS).  Eligible patients in the …

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