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Alnylam Announces U.S. Food and Drug Administration Has Granted Priority Review of the Lumasiran New Drug Application for the Treatment of Primary Hyperoxaluria Type 1
— PDUFA Date Set for December 3, 2020 — CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n— PDUFA Date Set for December 3, 2020 —\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the U.S. Food and Drug Administration (FDA) has accepted the Company’s New Drug Application (NDA) for lumasiran, an investigational RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – in development for the treatment of primary hyperoxaluria type 1 (PH1). The FDA also granted Priority Review for the NDA, a designation for medicines that have the potential to provide significant improvements in the treatment, prevention or diagnosis of a serious disease, with the goal of FDA taking action within six months compared to 10 months under standard review.\n\n\n“We are pleased to have received Priority Review status for lumasiran and look forward to continuing to work closely with the FDA during the review process,” said Pritesh J. Gandhi, PharmD., Vice President and General Manager, Lumasiran Program at Alnylam. “Award of Priority Review status adds to the previous grants of Breakthrough Therapy and Pediatric Rare Disease Designations. Together, we believe these underscore the potential of lumasiran to address the underlying pathophysiology of PH1 and the urgent need for an FDA-approved treatment for this progressive, devastating disease where liver transplantation is currently the only treatment that addresses the root cause of disease.”\n\n\nThe FDA has set an action date of December 3, 2020 under the Prescription Drug User Fee Act (PDUFA), and the Agency has indicated that they are not currently planning an advisory committee meeting as part of the NDA review.\n\n\nIn addition, the Marketing Authorisation Application (MAA) for lumasiran has been submitted to and validated by the European Medicines Agency (EMA). Lumasiran was previously granted an accelerated assessment by the EMA, which is awarded to medicines deemed to be of major public health interest and therapeutic innovation, and is designed to bring new treatments to patients more quickly. Accelerated assessment potentially reduces the Agency’s evaluation time from 210 to 150 days.\n\n\nLumasiran has also previously received Orphan Drug Designations for the treatment of PH1 in the U.S. and Europe and has received a Priority Medicines (PRIME)...