Alnylam's Onpattro: Not Following FDA Guidance Amplifies Clinical Meaningfulness Concerns

 
• By Sarah Karlin-Smith
  
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Even if Alnylam can get a favorable advisory committee vote and is able sneak out an approval for Onpattro for ATTR-associated cardiomyopathy, its lack of data on hard clinical outcomes makes it unlikely to unseat Pfizer’s tafamidis for the rare disease.

image of the heart (organ)
FDA advisors will vote on a supplemental indication for Onpattro for the treatment of ATTR cardiomyopathy 13 September. • Source: Shutterstock

Alnylam Pharmaceuticals Inc.’s failure to heed the US Food and Drug Administration’s guidance for clinical outcome assessment (COA)-based endpoints appears to have added to the agency’s difficulty in determining whether the small treatment effect in the company’s sole trial of Onpattro (patisiran) for the treatment of ATTR-associated cardiomyopathy is clinically meaningful and warrants an agency approval.

FDA’s Cardiovascular and Renal Drugs Advisory Committee will vote on 13 September on whether the benefits of Onpattro outweigh its...

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