Alnylam Submits Applications In U.S, EU To Support Label Expansion For Oxlumo For PH1 Treatment

(RTTNews) - Alnylam Pharmaceuticals Inc. (ALNY) said that it has submitted a supplemental New Drug Application or sNDA to the U.S. Food and Drug Administration and Type II Filing Variation to the European Medicines Agency for lumasiran, an investigational RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) - the gene encoding glycolate oxidase (GO) - for the reduction of plasma oxalate in the treatment of patients with advanced primary hyperoxaluria type 1 (PH1).

The applications are based on positive results from the ILLUMINATE-C single-arm, open-label Phase 3 study of lumasiran in patients of all ages with advanced PH1.

The phase 3 study showed substantial reductions in plasma oxalate in PH1 patients with end-stage Kidney Disease, including patients on Hemodialysis.

Oxlumo is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.