European Commission approves Oxlumo an RNAi therapeutic, to treat primary hyperoxaluria type 1.- Alnylam Pharma

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, 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. PH1 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. The approval in the European Union is based on efficacy and safety findings from both the ILLUMINATE-A and ILLUMINATE-B Phase III studies of lumasiran. In the ILLUMINATE-A study conducted in adults and children six years or older, lumasiran achieved the primary endpoint with a 53 percent mean reduction in urinary oxalate relative to placebo and showed a 65 percent mean reduction in urinary oxalate relative to baseline. .