FDA official announcements and press releases

Alnylam's Vutrisiran Achieves Positive Results in Phase 3 Study for ATTR-CM, Shares Surge 39%

Jun 24, 2024, 12:28 PM

Alnylam Pharmaceuticals reported positive topline results from its HELIOS-B Phase 3 study of vutrisiran, an investigational RNAi therapeutic for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). The study achieved statistical significance on primary and all secondary endpoints in both overall and monotherapy populations, with a P-value around 0.01. Vutrisiran met its main goal of reducing deaths and recurrent cardiovascular events, paving the way for it to become the first RNA silencing therapy on the market for ATTR amyloidosis with cardiomyopathy. The results are seen as a significant advancement in the treatment of this rare heart condition, potentially creating a multibillion-dollar opportunity for Alnylam. Following the announcement, Alnylam's shares surged up to 39% in pre-market trading, eventually rising by 33% to 38%. The company plans to file for US approval later this year.