Press release
Alnylam Submits Marketing Authorization Application to the European Medicines Agency for Investigational Vutrisiran for the Treatment of Hereditary ATTR Amyloidosis with Polyneuropathy
− If Approved, Vutrisiran will Provide a New, Subcutaneously Administered, Once-Quarterly Treatment Option for Patients with hATTR Amyloidosis − CAMBRIDGE,
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n− If Approved, Vutrisiran will Provide a New, Subcutaneously Administered, Once-Quarterly Treatment Option for Patients with hATTR Amyloidosis −\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for vutrisiran, an investigational RNAi therapeutic for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adult patients with polyneuropathy.\n\n“hATTR amyloidosis is a rare, rapidly progressive, debilitating and fatal condition and we are delighted that the EMA has agreed to review the regulatory submission for vutrisiran based on the positive 9-month data from the HELIOS-A Phase 3 study,” said Rena Denoncourt, Vice President, TTR Franchise Lead. “If approved, we believe that vutrisiran will provide an important new subcutaneously administered, once-quarterly treatment option for patients, with the potential to reverse manifestations of the disease in hATTR amyloidosis patients with polyneuropathy. We look forward to working closely with the EMA to bring vutrisiran to the hATTR amyloidosis community in Europe.”\n\nPositive 9-month results from the HELIOS-A Phase 3 study of vutrisiran were presented in April 2021 at the American Academy of Neurology (AAN) Virtual Annual Meeting. At 9 months, vutrisiran met the primary and all secondary endpoints, with statistically significant improvements in neuropathy, quality of life, and gait speed, and demonstrated an encouraging safety profile, relative to the external placebo group of the APOLLO study of patisiran. As aligned with the EMA, the results of the 18-month analysis from the HELIOS-A study will be provided to the Agency during its evaluation of the MAA.\n\nVutrisiran has been granted Orphan Drug Designation in the European Union (EU) and U.S. for the treatment of ATTR amyloidosis. In June 2021, the U.S. Food and Drug Administration (FDA) accepted the Company’s New Drug Application (NDA) submission for review for vutrisiran, with an action date set for April 14, 2022 under the Prescription Drug User Fee Act (PDUFA). The Company also plans to submit regulatory filings in Brazil and Japan in 2021.\n\nAbout Vutrisiran\n\nVutrisiran is an investigational, subcutaneously adminis...