ALN GO1 filed with EMA and FDA for primary hyperoxaluria type 1.- Alnylam Pharma

Alnylam Pharmaceuticals announced positive Phase III results from the ILLUMINATE-A study of ALN GO1 (lumasiran), an investigational RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – in development for the treatment of primary hyperoxaluria type 1 (PH1). Lumasiran achieved the ILLUMINATE-A primary endpoint with a 53.5 percent mean reduction in urinary oxalate relative to placebo (p=1.7×10-14) and showed a 65.4 percent mean reduction in urinary oxalate relative to baseline. All tested study secondary endpoints were met, including the proportion of patients achieving near-normalization (84 percent) or normalization (52 percent) of urinary oxalate, compared with zero percent in the placebo group. Lumasiran administration was associated with an encouraging safety and tolerability profile, with no serious or severe adverse events (AEs) and with mild injection site reactions (ISRs) as the most common drug-related AE. The clinical data were presented at a late-breaking session at the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) International Congress.

Based on the ILLUMINATE-A results, Alnylam filed a New Drug Application (NDA) with the FDA which has granted a Priority Review for the NDA and has set an action date of December 3, 2020 under the Prescription Drug User Fee Act. In addition, the Marketing Authorisation Application (MAA) for lumasiran has been submitted to and validated by the European Medicines Agency, and has received Accelerated Assessment designation.