Alnylam’s Onpattro Gets CRL, Company to Pull Expanded Indication

Alnylam Pharmaceuticals today announced that the FDA has issued a complete response letter (CRL) on its supplemental New Drug Application (sNDA) for Onpattro (patisiran) for the treatment of cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis and will no longer pursue the expanded indication.

The FDA issued the CRL despite recent recommendation from the Cardiovascular and Renal Drugs Advisory Committee who voted 9-3 on Sept. 13 that the benefits outweighed its risks for the new indication.

The company said the FDA had questioned the “clinical meaningfulness of patisiran’s treatment effects for the cardiomyopathy of ATTR amyloidosis,” and therefore “the sNDA for patisiran could not be approved in its present form.” Alnylam added that the FDA’s letter “did not identify any issues with respect to clinical safety, study conduct, drug quality or manufacturing.”

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