CNS - FDA accepts Alnylam's vutrisiran application for hereditary ATTR amyloidosis

Alnylam Pharmaceuticals (ALNY) announces that the FDA has accepted its New Drug Application ((NDA)) for vutrisiran, an investigational RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.The FDA has set PDUFA target action date of April 14, 2022, and the Agency has indicated that they are not currently planning an advisory committee meeting as part of NDA review. The NDA for vutrisiran was based on 9-month results from the Phase 3 HELIOS-A study. hATTR amyloidosis is an inherited, progressively debilitating, and fatal disease caused by variants in the TTR gene.

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FDA accepts Alnylam's vutrisiran application for hereditary ATTR amyloidosis