FDA accepts Alnylam’s sNDA for vutrisiran for ATTR-CM treatment

Alnylam (ALNY) Pharmaceuticals announced that the U.S. Food and Drug Administration or FDA, has accepted for review the Company’s supplemental New Drug Application sNDA for vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy ATTR-CM . Based on the Company’s use of a Priority Review Voucher, the FDA has set an action date goal of March 23 under the Prescription Drug User Fee Act PDUFA . The FDA has informed the Company that it is not planning to hold an advisory committee meeting at this time to review the application. Vutrisiran is the generic name for AMVUTTRA which is currently approved by the FDA for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis hATTR-PN in adults. “We are pleased that the FDA has accepted our sNDA for vutrisiran for the treatment of ATTR with cardiomyopathy – a steadily progressing, debilitating and ultimately fatal disease,” said Pushkal Garg, M.D., Chief Medical Officer of Alnylam. “In HELIOS-B, treatment with vutrisiran improved cardiovascular outcomes, survival, disease progression and quality of life, as compared to placebo, in a population reflective of today’s patients on substantial background treatment. We look forward to working with the FDA to support their review of the application and bring vutrisiran to patients with ATTR-CM in the U.S. early next year.”