CHMP recommends approval of Oxlumo for primary hyperoxaluria type 1.- Alnylam Pharmaceuticals

Alnylam Pharmaceuticals 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 ALN GO1 (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.The positive opinion is based on efficacy and safety findings of Oxlumo in PH1 patients, including data from both the ILLUMINATE-A and ILLUMINATE-B Phase III studies. Key primary and secondary endpoints included the reduction of urinary and plasma oxalate, and the proportion of patients achieving normalization or near-normalization of urinary oxalate in response to Oxlumo relative to placebo. Findings from the ILLUMINATE-A pivotal study were presented in June 2020 at the virtual European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) International Congress. Topline results from the ILLUMINATE-B pediatric study were reported in September; results from the primary analysis will be presented at the upcoming virtual American Society of Nephrology (ASN) Annual Congress on October 22.