Press release
Alnylam Submits Supplemental New Drug Application (sNDA) to U.S. Food and Drug Administration (FDA) for ONPATTRO® (patisiran) for the Treatment of the Cardiomyopathy of ATTR Amyloidosis
– sNDA Submission is Based on Findings from the APOLLO-B Phase 3 Study That Showed Patisiran Demonstrated Significant Improvement on Functional Capacity,
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n– sNDA Submission is Based on Findings from the APOLLO-B Phase 3 Study That Showed Patisiran Demonstrated Significant Improvement on Functional Capacity, Health Status and Quality of Life Compared to Placebo at Month 12 –\n\n– Study Also Demonstrated Encouraging Safety Profile in Patients with Cardiomyopathy of ATTR Amyloidosis –\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the submission of its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy. 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.\n\n“We are excited by today’s announcement as the submission of our sNDA brings us one step closer to making patisiran available to patients with ATTR amyloidosis with cardiomyopathy. Patisiran has the potential to improve the functional capacity and quality of life of patients with ATTR amyloidosis with cardiomyopathy, an increasingly recognized cause of heart failure for which there are limited treatment options,” said Rena N. Denoncourt, Vice President, TTR Franchise Lead. “The positive results of the APOLLO-B Phase 3 study of patisiran validate the therapeutic hypothesis that TTR silencing by an RNAi therapeutic may be an effective approach to treating cardiomyopathy of both wild-type and hereditary ATTR amyloidosis. This is an important milestone as we work to build an industry-leading franchise for the treatment of ATTR amyloidosis.”\n\nThe application to the FDA was based on positive results from APOLLO-B, a Phase 3, randomized, double-blind, placebo-controlled multicenter global study that demonstrated the effects of patisiran on functional capacity and quality of life in patients with ATTR amyloidosis with cardiomyopathy. The safety profile in APOLLO-B was consistent with what was observed in APOLLO and in postmarketing use of ONPATTRO. In APOLLO-B, the majority of adverse events (AEs) were mild or moderate in severity. The 12-month results from the study were presented at the 18th International Symposium on Amyloidosis (ISA) ...