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Alnylam: HELIOS-A Phase 3 Study Of Vutrisiran Met Primary And Secondary Endpoints
Vutrisiran is an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis.
Alnylam said that based on these positive results, it plans to submit a New Drug Application or NDA for vutrisiran with the U.S. Food and Drug Administration (FDA) in early 2021, and to follow with regulatory filings in additional countries, such as Brazil and Japan.
The primary endpoint of the HELIOS-A Phase 3 trial was the change from baseline in the modified Neuropathy Impairment Score (mNIS+7) at 9 months as compared to historical placebo data from the APOLLO Phase 3 study of patisiran.