CAMBRIDGE, Mass.– Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the Company will present full 18-month results from the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis, at the Société Francophone du Nerf Périphérique (SFNP) Annual Meeting. The meeting will be held January 21-22 in Paris, France, and convenes healthcare professionals focused on the peripheral nervous system. The Company previously announced positive topline 18-month results from the HELIOS-A study in October 2021. David Adams M.D., Ph.D., Department of Neurology, Coordinator of the National Reference Center for Familial Amyloid Polyneuropathy (FAP) and Rare Neuropathies, Bicêtre Hospital, Greater Paris University Hospitals, AP-HP, will present “HELIOS-A: Study of Vutrisiran in Patients with hATTR Amyloidosis” on January 21, 2022.
Vutrisiran is under review by the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), the Brazilian Health Regulatory Agency (ANVISA), and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). Vutrisiran has been granted Orphan Drug Designation in the U.S. and the European Union (EU) for the treatment of ATTR amyloidosis. Vutrisiran has also been granted a Fast Track designation in the U.S. for the treatment of the polyneuropathy of hATTR amyloidosis in adults. In the U.S., vutrisiran has received an action date under the Prescription Drug User Fee Act (PDUFA) of April 14, 2022. The Company received Orphan Drug Designation in Japan for transthyretin type familial amyloidosis with polyneuropathy.
