RNAi therapeutics company Alnylam Pharmaceuticals Inc (Nasdaq: ALNY) on Monday announced FDA acceptance of its supplemental New Drug Application (sNDA) for vutrisiran, an investigational RNAi therapeutic for ATTR amyloidosis with cardiomyopathy (ATTR-CM). Leveraging a Priority Review Voucher, the Prescription Drug User Fee Act (PDUFA) goal date is 23 March 2025. No advisory committee meeting is planned for the review.

Vutrisiran, marketed as AMVUTTRA, is already FDA-approved for hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy. If approved, it would be the first US treatment targeting both polyneuropathy and cardiomyopathy in transthyretin amyloidosis (ATTR).

The sNDA is supported by positive Phase 3 HELIOS-B trial data, showing significant benefits in survival, cardiovascular outcomes and quality of life for ATTR-CM patients. Results were consistent with the established safety profile and published in The New England Journal of Medicine on 30 August 2024.

Alnylam continues to expand its pipeline of transformative medicines addressing unmet medical needs globally.