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Alnylam Receives Approval for OXLUMO™ (lumasiran) in the European Union for the Treatment of Primary Hyperoxaluria Type 1 in All Age Groups
– OXLUMO is the First Therapeutic Approved for the Treatment of PH1, and the Only Therapy Proven to Lower Harmful Oxalate Levels that Drive the Progression
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n– OXLUMO is the First Therapeutic Approved for the Treatment of PH1, and the Only Therapy Proven to Lower Harmful Oxalate Levels that Drive the Progression of PH1 Disease –\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the European Commission (EC) has granted marketing authorization for OXLUMO™ (lumasiran), an RNAi therapeutic, for the treatment of primary hyperoxaluria type 1 (PH1) in all age groups.\n\nPH1 is an ultra-rare orphan disease characterized by excessive oxalate production, which can lead to life threatening end-stage renal disease (ESRD) and other systemic complications. Heterogeneity in disease manifestation often contributes to delays in diagnosis – particularly in adult PH1 patients, with a median time from symptoms onset to diagnosis of approximately six years. Untreated PH1 leads to progressive kidney damage; patients with advanced kidney disease require intensive dialysis to help filter waste products, including oxalate, from their blood until they are able and eligible to receive a dual or sequential liver/kidney transplant, an invasive procedure associated with a high risk of morbidity and mortality, and life-long immunosuppression.\n\n“Prior to now there have been no approved treatment options for PH1 in Europe, so this is a potentially life-changing milestone for people diagnosed with this ultra-rare, debilitating disease - many of whom are infants and children - and their families. Lumasiran will address the urgent unmet need that exists for patients with PH1 and its approval today marks our continued commitment to rare disease communities,” said John Maraganore, Ph.D., Chief Executive Officer, Alnylam Pharmaceuticals. “Alnylam has taken lumasiran from identification of compound to regulatory approval in just six years and we will progress with the same sense of urgency as we work with national reimbursement bodies across Europe to bring lumasiran to patients.”\n\nLumasiran is an RNAi therapeutic targeting the hydroxyacid oxidase 1 (HAO1) mRNA that encodes glycolate oxidase (GO) – an enzyme upstream of the disease-causing defect in PH1. By degrading the HAO1 mRNA and reducing the synthesis of GO, lumasiran stops the production of oxalate – the toxic metabolite that directly contributes to the clinical ma...