: Alnylam to scrap sNDA for patisiran after receiving FDA complete response letter

Alnylam Pharmaceuticals Inc. ALNY said Monday it would no longer pursue its supplemental new drug application (sNDA) for patisiran as a treatment for the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis after receiving a complete response letter from the U.S. Food and Drug Administration. Patisiran is already approved for the treatment of the polyneuropathy of hereditary ATTR amyloidosis in adults. The CRL, which means the FDA cannot approve the sNDA for now, does not impact the other indication. “The CRL indicated that the clinical meaningfulness of patisiran’s treatment effects for the cardiomyopathy of ATTR amyloidosis had not been established, and therefore, the sNDA for patisiran could not be approved in its present form,” the company said in a statement. Alnylam will instead focus on the HELIOS-B Phase 3 study of vutrisiran, an investigational RNAi therapeutic subcutaneously administered once every three months in development for the treatment of the cardiomyopathy of ATTR amyloidosis, and ALN-TTRsc04, which is dosed annually. Transthyretin-mediated (ATTR) amyloidosis is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart and gastrointestinal tract, said the statement. The stock was halted premarket for the news.

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