The US Food and Drug Administration (FDA) has accepted a supplemental New Drug Application (sNDA) for patisiran for the treatment of cardiomyopathy in transthyretin amyloidosis (ATTR). The drug is currently approved by the FDA to treat polyneuropathy in adults with hereditary ATTR (hATTR).
“ATTR amyloidosis with cardiomyopathy is an increasingly recognized cause of heart failure for which there are limited treatment options,” Rena N. Denoncourt, vice president and TTR franchise lead at Alnylam Pharmaceuticals, said in a press release.
“The FDA’s acceptance of our sNDA for patisiran is a positive step forward as we work to bring patients with ATTR amyloidosis with cardiomyopathy a new treatment option that addresses the underlying cause of disease and has the potential to meaningfully improve functional capacity and quality of life.”
The application was based on positive results from the APOLLO-B trial—a randomized, double-blind, placebo-controlled, multicenter, global phase 3 study on the efficacy and safety of patisiran in participants with ATTR with cardiomyopathy. The 12-month data showed that patisiran improved the participants’ functional capacity, health status, and quality of life.
Read more about hATTR therapies
The results from APOLLO-B were presented at the 18th International Symposium on Amyloidosis (ISA) and the Heart Failure Society of America’s Annual Scientific Meeting in 2022.
The FDA has issued an action date of October 8, 2023, under the Prescription Drug User Fee Act. The agency has not identified any review issues and is planning to hold an advisory committee meeting to discuss the regulatory application, according to the press release from Alnylam.
Reference
Alnylam announces U.S. Food and Drug Administration (FDA) acceptance of supplemental New Drug Application for Onpattro® (patisiran) for the treatment of the cardiomyopathy of ATTR amyloidosis. News release. Alnylam Pharmaceuticals; February 21, 2023.
