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BioWorld - Tuesday, December 30, 2025
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Home » Alnylam pulls back on new Onpattro indication after surprise CRL
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Alnylam pulls back on new Onpattro indication after surprise CRL

Oct. 9, 2023
By Mari Serebrov
Following an unexpected FDA complete response letter (CRL), Alnylam Pharmaceuticals Inc. said it will no longer pursue an expanded indication for Onpattro (patisiran) in the U.S. The RNAi therapeutic was approved in 2018 to treat polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis and seemed well on its way to snagging a second U.S. indication after the FDA’s Cardiovascular and Renal Drugs Advisory Committee voted 9-3 in September that the drug’s benefits outweighed its risks as a treatment for cardiomyopathy of ATTR amyloidosis. The FDA disagreed with the committee, saying in the CRL that patisiran’s clinical meaningfulness had not been established in the proposed indication.
BioWorld Regulatory Endocrine/metabolic U.S. FDA

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