Alnylam submits regulatory application to EMA for vutrisiran for ATTR-CM

Alnylam Pharmaceuticals announced the submission of a Type II Variation to the European Medicines Agency EMA for vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy ATTR-CM. Vutrisiran is the generic name for AMVUTTRA , which is currently approved in the European Union for the treatment of hereditary transthyretin-mediated or hATTR, amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy. “Today marks another important milestone in our journey to bring RNAi therapeutics to patients with high unmet need around the world,” said Pushkal Garg, M.D., Chief Medical Officer of Alnylam. “ATTR-CM is a rapidly progressive, debilitating, and life-threatening disease that is an increasingly recognized cause of heart failure. Vutrisiran rapidly knocks down TTR, and in the HELIOS-B study treatment with vutrisiran substantially reduced all-cause mortality and cardiovascular events, underscoring the potential of this therapy for those living with the disease. We look forward to working closely with the EMA with the aim to bring this new treatment option to patients as soon as possible.”