The FDA’s Cardiovascular and Renal Drugs Advisory Committee will meet tomorrow to discuss the efficacy of Alnylam’s Onpattro (patisiran) for the treatment of cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults.
While Alnylam has touted the treatment’s statistical significance in this setting, hitting its primary endpoint and meeting its first secondary endpoint in a pivotal trial, the FDA is raising questions on the effect size. If approved in this indication, Onpattro would have to compete with Pfizer’s Vyndamax (tafamidis) capsules, which FDA approved in 2019 as the first treatment for ATTR-CM.
“Despite achievement of statistical significance, the change from baseline at Month 12 in 6MWT [6-Minute Walk Test] was small,” the FDA said in briefing documents released yesterday ahead of the meeting. “Approval of [Pfizer’s] tafamidis markedly improved the treatment of patients with ATTR-CM (wild-type and variant), and it is now the standard of…
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