Alnylam Pharmaceuticals

Huntington’s disease, caused by a defective gene, leads to protein clumps damaging brain cells. A new trial tests ALN-HTT02, an RNAi therapeutic, aiming to reduce the damaging protein. Up to 54 participants will receive either the drug or a placebo, with the option to switch. The trial, led by Professor Sarah Tabrizi, seeks to slow disease progression and improve patient outcomes.

DelveInsight's 'Hemophilia B Pipeline Insight 2024' report details 20+ pipeline drugs from 15+ companies, focusing on mechanism of action, route of administration, and molecule types. Key companies include Belief Biomed, TiumBio, and Pfizer, with therapies in various clinical stages. Notable recent developments include Novo Nordisk and ApcinteX trials in May 2024.

Sarepta Therapeutics announced a deal with Arrowhead Pharmaceuticals, acquiring four clinical-stage treatments and three preclinical assets for $500 million upfront and $325 million in shares. Arrowhead will complete Phase 1/2 trials before handing over development to Sarepta, which also gains rights to nominate six additional drug targets. The deal is seen as complementary to Sarepta's genetic medicine focus and aims to address concerns over its thin pipeline.

Alnylam Pharmaceuticals' vutrisiran sNDA for ATTR-CM accepted by FDA, with an action date of March 23, 2025. Vutrisiran, a generic of AMVUTTRA, treats ATTR by reducing transthyretin. HELIOS-B study showed vutrisiran improved cardiovascular outcomes, survival, and quality of life. Adverse reactions include pain, arthralgia, dyspnea, and vitamin A decrease, requiring supplementation. BridgeBio Pharma's Attruby also approved for ATTR-CM on Nov. 22, 2024.

The FDA accepted Alnylam's sNDA for vutrisiran to treat ATTR-CM, setting a PDUFA date of March 23, 2025. Vutrisiran, already approved for hATTR-PN, showed significant risk reduction in mortality and CV events in the HELIOS-B trial, with favorable effects on functional capacity and quality of life. Alnylam anticipates vutrisiran could be the first to treat both hATTR polyneuropathy and ATTR cardiomyopathy.

BridgeBio’s Attruby, an oral drug for transthyretin amyloid cardiomyopathy (ATTR-CM), has been approved by the FDA, priced at $18,759.12 for a 28-day supply. It significantly improves survival and reduces heart disease-related hospitalizations but lacks a mortality benefit. BridgeBio partners with Bayer for European marketing and AstraZeneca’s Alexion for Japan. Analysts predict limited uptake due to Pfizer’s established Vyndaqel, but potential for $2.5 billion in global sales by 2035.

Hemophilia B market to grow significantly by 2034, driven by gene therapies like HEMGENIX and BEQVEZ, priced at $3.5 million each. Key companies include Centessa Pharmaceuticals, Sanofi, Pfizer, and Novo Nordisk. Market dynamics influenced by increasing prevalence, prophylactic treatment focus, and pipeline advancements.

BridgeBio Pharma's acoramidis (Attruby) wins FDA approval for treating ATTR cardiomyopathy, competing with Pfizer's tafamidis. Attruby, a near-complete TTR stabilizer, aims to reduce cardiovascular death and hospitalization. Despite initial trial setbacks, 30-month data supported approval, positioning Attruby as the first oral stabilizer with near-complete stabilization claims. BridgeBio estimates a $15-20 billion market, dominated by Pfizer, with Alnylam and AstraZeneca also entering the space. Attruby's pricing is competitive but above cost-effectiveness benchmarks, with ongoing European review and a Bayer licensing deal.

Trump's health nominees, including CDC's Dave Weldon and FDA's Marty Makary, could bring significant changes. Merck's Winrevair showed positive results in a Phase 3 trial, while Cassava's sumifilam failed in Alzheimer's treatment. FDA approved BridgeBio's Attruby for ATTR-CM, priced lower than Pfizer's drugs. Singulair linked to mental health issues. Sarclisa trials highlighted racial disparities in clinical trial composition.

Alnylam announced FDA acceptance of vutrisiran's sNDA for ATTR-CM, with a PDUFA date of March 23, 2025. The application is based on HELIOS-B Phase 3 study results showing improved cardiovascular outcomes, survival, and quality of life in ATTR-CM patients. If approved, vutrisiran would be the first U.S. treatment for both polyneuropathy and cardiomyopathy manifestations of ATTR amyloidosis.