Alnylam Onpattro’s Expanded Use Gets Weak Thumbs Up From US FDA Panel

Sep 14 2023

• By Sarah Karlin-Smith

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A restrictive label could be the result of the lukewarm endorsement from the majority of the agency’s Cardiovascular and Renal Drugs Advisory Committee for use of Onpattro for the treatment of ATTR-associated cardiomyopathy.

baseball player bunting — Two FDA advisors described Alnylam’s Onpattro ATTR cardiomyopathy data as a 'bunt single' in lukewarm positive panel. • Source: Shutterstock

US Food and Drug Administration advisors voted 9-3 that the benefits of Alnylam Pharmaceuticals Inc.’s Onpattro outweigh its risk for the treatment of ATTR-associated cardiomyopathy, but the seemingly lopsided vote was not overly enthusiastic and may suggest the drug is headed for a limited use label or perhaps still the complete response letter FDA seemed like it might have been leaning towards going into the 13 September meeting.

Alnylam is seeking a supplemental indication for Onpattro (patisiran) for the treatment of cardiomyopathy of wild type or inherited transthyretin...

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