FDA posts briefing document ahead of panel meeting on Alnylam’s patisiran

Yesterday, the FDA posted briefing documents ahead an Advisory Committee meeting being convened on September 13 to discuss whether Alnylam’s Onpattro, or patisiran, has clinically meaningful benefits for the treatment of cardiomyopathy of wild-type transthyretin mediated or hereditary transthyretin-mediated amyloidosis. The document prepared by the FDA staff states in part: “In summary, APOLLO-B was a well-designed and executed clinical trial meeting its pre-specified objectives. Although the primary efficacy endpoint of 6MWT and key secondary endpoint of KCCQ-OSS were statistically significant, the effects of patisiran compared to placebo on both endpoints were small, of questionable clinical meaningfulness, and may not be detectable by patients . Moreover, the effects of patisiran compared to placebo on 6MWT appeared confined to patients not on background therapy with tafamidis.”