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Alnylam Receives Positive CHMP Opinion for OXLUMO™ (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1 in All Age Groups
– Positive Opinion is Based on Results from ILLUMINATE-A and ILLUMINATE-B Phase 3 Studies – – European Commission Decision on Approval Expected in Q4 2020 –
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n– Positive Opinion is Based on Results from ILLUMINATE-A and ILLUMINATE-B Phase 3 Studies – \n\n– European Commission Decision on Approval Expected in Q4 2020 – \n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending approval of lumasiran, an investigational RNAi therapeutic targeting the hydroxyacid oxidase 1 (HAO1) mRNA – encoding glycolate oxidase (GO) – in development for the treatment of primary hyperoxaluria type 1 (PH1). If approved by the European Commission (EC), lumasiran will be marketed in Europe under the brand name OXLUMO™.\n\nPH1 is an ultra-rare orphan disease characterized by excessive oxalate production, which can lead to end-stage renal disease (ESRD) and other systemic complications. PH1 affects approximately 3.5 to 4 individuals per million in Europe and the United States. Heterogeneity in disease manifestation often contributes to delays in diagnosis, with a median time to diagnosis of approximately six years. PH1 leads to progressive kidney damage, and patients with advanced kidney disease require intensive dialysis to help filter waste products 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“This positive CHMP opinion recognizes the potential of OXLUMO to address the urgent unmet need that exists for patients of all ages impacted by primary hyperoxaluria type 1. Since there are no approved pharmacologic treatment options for PH1, we believe this is very encouraging news for those living with this ultra-rare, potentially life-threatening disease and for their families,” said Brendan Martin, Acting Head of Europe, Canada, Middle East and Africa (CEMEA), Alnylam Pharmaceuticals.\n\n“People living with PH1 experience a progressive decline in kidney function due to the overproduction of oxalate, which can lead to end-stage renal disease. Current treatment approaches aim to delay progression to renal failure but do not prevent oxalate overproduction,” said Pushkal Garg, M.D., Chief Medical Officer, Alnylam Ph...