US FDA Rejects Onpattro Label Expansion: History Repeats In Reverse?

In going against an advisory committee vote, the agency produces ironic echoes with the history of the approved treatment for ATTR-CM, Pfizer’s Vyndaqel.

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The US Food & Drug Administration’s rejection of a broader label for Alnylam Pharmaceuticals Inc.’s Onpattro (patisiran) is yet another reminder of the surprising intricacies of product assessments at the agency.

Less than a month ago, the Cardio-Renal Drugs Advisory Committee voted 9-3 that the benefits of the drug for use in AATR-associated cardiomyopathy outweighed the risks. The committee’s enthusiasm for the efficacy of Onpattro was lukewarm at best – but the vote was still a noteworthy endorsement given the bluntly negative FDA review of the data presented during the meeting

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