Alnylam Pharmaceuticals announced the submission of its supplemental New Drug Application to the U.S. Food and Drug Administration for patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated 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. "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."
Published first on TheFly
