US FDA Expands Approval for Alnylam's Amvuttra to Treat Rare Heart Disease

On March 20, 2025, the U.S. FDA approved Alnylam’s RNAi therapeutic Amvuttra (vutrisiran) for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM), a rare and deadly heart disease. The approval expands the drug’s indication from its 2022 use for treating nerve damage associated with ATTR amyloidosis, making it the first treatment available for both neuropathy and cardiomyopathy caused by the disease. This decision was backed by statistically significant results from the HELIOS-B Phase 3 clinical trial.

Alnylam aims to leverage this success for profitability and further investment in future treatments. With the market for ATTR-CM therapies expected to reach $11 billion by 2032 and Amvuttra sales projected at $5 billion by 2029, this milestone positions the company strongly against competitors like Pfizer's Vyndaqel. Priced at $119,351 per prefilled syringe administered quarterly, Amvuttra offers a promising new therapeutic strategy for patients.