1. Vutrisiran, an RNA interference therapeutic, has been approved by the FDA for treating cardiomyopathy associated with transthyretin-mediated amyloidosis.
2. It reduces the risk of cardiovascular death, hospital admissions, and urgent heart failure visits.
3. Vutrisiran is the first approved treatment addressing both cardiomyopathy and polyneuropathy manifestations of ATTR amyloidosis.
4. The treatment achieves statistical significance compared to placebo in reducing mortality and cardiovascular events.
5. It is given subcutaneously every 3 months and is associated with preservation of functional capacity and quality of life.
6. No new safety concerns were identified in the trial.
7. Common adverse events included extremity pain, arthralgia, dyspnea, and decreased vitamin A levels.

Source: Alnylam Pharmaceuticals