Alnylam Gets OK for Application for ONPATTRO® (patisiran) for Cardiomyopathy of ATTR Amyloidosis

 -Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the Company’s supplemental New Drug Application (sNDA) for patisiran, an investigational RNAi therapeutic in development for the treatment of the cardiomyopathy of transthyretin-mediated (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 U.S. 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."

https://finance.yahoo.com/news/alnylam-announces-u-food-drug-130000470.html