Press release
Alnylam Announces U.S. Food and Drug Administration Acceptance of Supplemental New Drug Application for OXLUMO® for the Treatment of Advanced Primary Hyperoxaluria Type 1
PDUFA Date Set for October 6, 2022 CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company,
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\nPDUFA Date Set for October 6, 2022\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 supplemental New Drug Application (sNDA) for lumasiran, an investigational RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – for the reduction of plasma oxalate in the treatment of patients with advanced primary hyperoxaluria type 1 (PH1). The FDA has set an action date of October 6, 2022, under the Prescription Drug User Fee Act (PDUFA).\n\n“We are pleased that the FDA has accepted our sNDA for lumasiran based on the positive six-month results of the ILLUMINATE-C study showing that lumasiran can substantially reduce plasma oxalate levels in patients with compromised renal function due to PH1, including those on hemodialysis,” said Pushkal Garg, MD., Chief Medical Officer and EVP, Clinical Development and Medical Affairs at Alnylam. “This filing acceptance is a positive step for patients with advanced PH1, who are at risk for the devastating complications of systemic oxalosis.”\n\nAdditionally, a Type II Variation for lumasiran to amend the label to further inform on the use of lumasiran in patients with advanced PH1 was submitted to and validated by the European Medicines Agency (EMA) in December 2021. The application is undergoing review by the Committee for Medicinal Products for Human Use (CHMP), which will then issue an opinion to the European Commission.\n\nIn 2020, OXLUMO was approved by the FDA for the treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients and by the EMA for the treatment of PH1 in all age groups.\n\nOXLUMO® (lumasiran) INDICATION AND IMPORTANT SAFETY INFORMATION\n\nIndication\n\nOXLUMO is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.\n\nImportant Safety Information\n\nAdverse Reactions\n\nThe most common adverse reaction that occurred in patients treated with OXLUMO was injection site reaction (38%). Symptoms included erythema, pain, pruritus, and swelling.\n\nPregnancy and Lactation\n\nNo data are available on the use of OXLUMO in pregnant women. No data are available on the presence of OXLUMO...