FDA

Amvuttra approved for transthyretin amyloidosis cardiomyopathy

March 31, 2025

FDA approved Amvuttra (vutrisiran) for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations, and urgent heart failure visits.

Efficacy

Efficacy was evaluated in a multicenter, international, randomized, double-blind, placebo-controlled trial (HELIOS-B, NCT04153149) in 654 adult patients with wild-type or hereditary ATTR-CM. Patients were randomized 1:1 to receive 25 mg of Amvuttra (n=326) SC q3mo, or matching placebo (n=328). The primary efficacy endpoint was the composite outcome of all-cause mortality and recurrent CV events (CV hospitalizations and urgent heart failure visits) during the double-blind treatment period of up to 36 months, evaluated in the overall population and in the monotherapy population (patients not receiving tafamidis at study baseline). Amvuttra led to significant reduction in the risk of all-cause mortality and recurrent CV events compared with placebo in the overall and monotherapy population of 28% and 33%, respectively. Both components of the primary composite endpoint individually contributed to the treatment effect in the overall and monotherapy population.

Safety

The most common adverse reactions (≥5%) were pain in extremity, arthralgia, dyspnea, and vitamin A decreased.

Sources:

Amvuttra (vutrisiran) [package insert]. U.S. Food and Drug Administration. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/215515s006lbl.pdf Revised March 2025. Accessed March 27, 2025.

Alnylam announces FDA approval of Amvuttra® (vutrisiran), the first RNAi therapeutic to reduce cardiovascular death, hospitalizations and urgent heart failure visits in adults with ATTR amyloidosis with cardiomyopathy (ATTR-CM). [News release]. 2025. https://investors.alnylam.com/press-release?id=28831