A phase III trial shows that vutrisiran, an RNA interference therapeutic, has been approved by the FDA for treating cardiomyopathy associated with transthyretin-mediated amyloidosis.

This therapy reduces the risk of cardiovascular death, hospital admissions, and urgent heart failure visits, making it the first approved treatment addressing both cardiomyopathy and polyneuropathy manifestations of ATTR amyloidosis. Vutrisiran reduces transthyretin production, achieving statistical significance compared to placebo in reducing mortality and cardiovascular events. The treatment is given subcutaneously every 3 months and is associated with preservation of functional capacity, quality of life, and improvements in cardiac stress biomarkers. No new safety concerns were identified in the trial.

Source: Alnylam Pharmaceuticals