Alnylam is seeking approval for Onpattro’s use in cardiomyopathy related to transthyretin-mediated (ATTR) amyloidosis. The FDA set an action date of Oct. 8, 2023.
The FDA has accepted Alnylam Pharmaceutical’s supplemental new drug application (sNDA) for Onpattro (patisiran), an RNAi therapeutic to treat the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis. ATTR amyloidosis is a rare, rapidly progressive, debilitating disease caused by misfolded transthyretin proteins that accumulate as amyloid fibrils in multiple tissues including the nerves, heart, and gastrointestinal tract.
ATTR amyloidosis with cardiomyopathy is an increasingly recognized cause of heart failure for which there are limited treatment options, Rena N. Denoncourt, vice president, TTR franchise lead for Alynlam, said in a press release.
The FDA has set an action date of Oct. 8, 2023, under the Prescription Drug User Fee Act (PDUFA). The agency has not identified any review issues and said it would hold an advisory committee meeting to discuss the application.
Onpattro, already approved by the FDA to treat the polyneuropathy of hereditary ATTR amyloidosis in adults – which affects the peripheral nerves — netted $558 million in revenue in 2022. Its wholesale acquisition cost is $9,785 per vial.
The sNDA was based on positive results from APOLLO-B, a randomized, double-blind, placebo-controlled, multicenter, global phase 3 study that demonstrated favorable effects of Onpattro on both functional capacity and quality of life in patients with ATTR amyloidosis with cardiomyopathy relative to placebo at 12 months. The study achieved its primary endpoint, with Onpattro demonstrating a statistically significant and clinically meaningful benefit on functional capacity, as measured by the 6-Minute Walk Test, compared with placebo, Alynlam said.
The study also met its first secondary endpoint, demonstrating a statistically significant and clinically meaningful benefit on health status and quality of life compared with placebo.
The majority of adverse events were mild or moderate in severity, and the overall safety profile in APOLLO-B was consistent with prior clinical trials and post-marketing experience with Onpattro.
The 12-month results were presented at the 18th International Symposium on Amyloidosis (ISA) on September 8, 2022 and at the Heart Failure Society of America’s Annual Scientific Meeting on Sept. 30, 2022.