FDA accepts sNDA for Onpattro for the treatment of the cardiomyopathy of ATTR amyloidosis

The FDA has set an action date of October 8, 2023 under the Prescription Drug User Fee Act (PDUFA).

In their file acceptance letter, the FDA stated that they have not identified any review issues. The Agency also noted that they are planning to hold an advisory committee meeting to discuss the application. Patisiran is the established name for Onpattro, which is currently approved by the FDA for the treatment of the polyneuropathy of hereditary ATTR amyloidosis in adults.

“ATTR amyloidosis with cardiomyopathy is an increasingly recognized cause of heart failure for which there are limited treatment options. 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,” said Rena N. Denoncourt, Vice President, TTR Franchise Lead. “The acceptance also marks another important milestone as we continue to build an industry-leading franchise for the treatment of ATTR amyloidosis.”

The application to the FDA was based on positive results from APOLLO-B, a randomized, double-blind, placebo-controlled, multicenter, global Phase III study that demonstrated favorable effects of patisiran on both functional capacity and quality of life in patients with ATTR amyloidosis with cardiomyopathy relative to placebo at 12 months. 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 from the study 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 September 30, 2022.