CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNA interference (RNAi) therapeutics company, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending approval of its RNAi therapeutic vutrisiran for the treatment of wild type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). ATTR-CM is a debilitating, rapidly progressive and potentially fatal disease for which there are limited treatment options. Vutrisiran is currently approved in the European Union (EU) under the brand name AMVUTTRA® for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy.
ATTR-CM is caused by the deposition of misfolded transthyretin (TTR) fibrils, which drive progressive and irreversible cardiovascular damage and premature death. Vutrisiran is an RNAi therapeutic that works upstream to reduce the production of TTR at its source, resulting in sustained TTR knockdown. In Europe, it is administered as a subcutaneous injection once every three months, either by a healthcare professional, or self-administered by patients or their caregivers, offering flexibility in treatment delivery. Vutrisiran has the potential to be the first and only RNAi therapeutic to receive European Commission approval for ATTR-CM, offering a clinically differentiated approach to the treatment of this disease.
“This positive CHMP opinion marks another important milestone in our efforts to bring vutrisiran to people around the world living with ATTR amyloidosis with cardiomyopathy,” said Pushkal Garg, M.D., Chief Medical Officer of Alnylam. “In the HELIOS-B study, vutrisiran treatment resulted in rapid knockdown of TTR and led to improved survival, fewer hospitalizations and less disease progression in patients with ATTR-CM, nearly half of whom were on a TTR stabilizer. Combined with its quarterly dosing and well-established safety profile, we believe vutrisiran could offer an important new treatment option for patients in the EU.”
The CHMP opinion was based on positive results from the pivotal HELIOS-B Phase 3, randomized, double-blind, placebo-controlled multicenter global study, which met all 10 of its pre-specified primary and secondary endpoints across both the overall and monotherapy populations. The findings demonstrated the benefits of vutrisiran on outcomes of mortality and cardiovascular events, as well as functional capacity (6-minute walk test), health status and quality of life (Kansas City Cardiomyopathy Questionnaire), and heart failure symptoms and severity (NYHA class) in patients with ATTR-CM, with consistent effects across all patient subgroups, including those on a concomitant TTR stabilizer. In HELIOS-B, rates of adverse events (AEs), serious AEs, severe AEs and AEs leading to study drug discontinuation were similar between the vutrisiran and placebo arms. The safety profile of vutrisiran is characterized by injection site reactions and increase in blood alkaline phosphatase and alanine transaminase. Detailed results from the HELIOS-B study were published in The New England Journal of Medicine.1
Vutrisiran was approved by the U.S. Food and Drug Administration (FDA) on 20 March 2025 and the Brazilian Health Regulatory Agency (ANVISA) on 31 March 2025 for the treatment of the cardiomyopathy of wild-type or hereditary ATTR-CM. It is currently under review by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). Alnylam remains on track to proceed with additional global regulatory submissions for vutrisiran in 2025 and beyond.