Alnylam Gets FDA Nod for Expanded Use of Amvuttra in Cardiomyopathy

Core Viewpoint - Alnylam Pharmaceuticals received FDA approval for the label expansion of its lead drug, Amvuttra, to treat cardiomyopathy associated with transthyretin-mediated amyloidosis, marking a significant milestone as it becomes the first and only approved therapy for both ATTR-CM and hATTR-PN in adults [1][3][4] Company Overview - Amvuttra is an RNAi therapeutic previously approved for treating polyneuropathy associated with transthyretin-mediated amyloidosis in adults [2] - The drug generated sales of $970.5 million in 2024, reflecting a 74% year-over-year increase, driven by new patient treatments and switches from Onpattro [11] Clinical Data and Efficacy - The approval is based on positive results from the pivotal phase III HELIOS-B study, which met all primary and secondary endpoints with statistical significance [4] - Amvuttra demonstrated a reduction in mortality and cardiovascular events, while improving functional capacity, quality of life, and heart failure symptoms in ATTR-CM patients [6] Safety Profile - The safety profile of Amvuttra in the HELIOS-B study was consistent with its known profile for treating hATTR amyloidosis, with similar rates of adverse events between Amvuttra and placebo groups [7] Market Potential - Amvuttra enjoys near-universal insurance coverage for hATTR-PN, and similar coverage is anticipated for ATTR-CM, supported by strong clinical data [8] - Regulatory applications for Amvuttra's ATTR-CM indication are under review in the EU, Brazil, and Japan, with further applications expected in 2025 [9][10] Growth Prospects - Alnylam believes that Amvuttra has the potential to become the new standard of care for ATTR-CM, which could significantly expand the eligible patient population and drive substantial growth for the company [10]