Alnylam's Amvuttra Secures FDA Approval For Expanded Use For Rare Heart Disease, Stock Jumps

Core Insights - The U.S. FDA approved Alnylam Pharmaceuticals' supplemental application for Amvuttra to treat adults with ATTR-CM, aiming to reduce cardiovascular mortality, hospitalizations, and urgent heart failure visits [1][2]. Group 1: Approval and Indications - Amvuttra is now the first and only FDA-approved therapy for both ATTR-CM and hATTR-PN in adults [2]. - ATTR-CM affects approximately 150,000 individuals in the U.S. and over 300,000 globally [2]. Group 2: Clinical Trial Results - The approval is based on the HELIOS-B Phase 3 clinical trial, which showed statistical significance on all 10 primary and secondary endpoints compared to placebo [3]. - Amvuttra reduced the risk of all-cause mortality and recurrent cardiovascular events by 28% during a treatment period of up to 36 months [3]. - Mortality was significantly reduced by 36% through 42 months in a secondary endpoint analysis [4]. - In the monotherapy population, Amvuttra reduced the risk of all-cause mortality and recurrent cardiovascular events by 33% and the risk of mortality by 35% through 42 months [4]. Group 3: Market Dynamics and Financials - Amvuttra in hATTR-PN is covered by insurers for around 99% of patients, with most paying nothing out-of-pocket, and similar coverage is expected for ATTR-CM [5]. - In 2024, Amvuttra sales reached $970.45 million, an increase from$557.84 million the previous year [6]. Group 4: Future Prospects - Marketing authorization applications based on HELIOS-B data are under review by several global health agencies, including the European Medicines Agency and the Brazilian Health Regulatory Agency [6]. - Alnylam plans to proceed with additional global regulatory submissions for vutrisiran in 2025 [6]. Group 5: Stock Performance - Following the approval news, ALNY stock rose by 8.46% to $274.98 [7].