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 risks for the treatment of cardiomyopathy of wild type or inherited transthyretin amyloidosis (ATTR)