FDA Approves Alnylam’s sNDA for Oxlumo in Advanced Primary Hyperoxaluria Type 1
The FDA has approved Alnylam Pharmaceuticals’ supplemental new drug application (sNDA) for Oxlumo (lumasiran) to treat primary hyperoxaluria type 1 by lowering plasma oxalate levels in adult and pediatric patients.
The drug was first approved in November 2020 to treat primary hyperoxaluria type 1 by lowering urinary oxalate in adult and pediatric patients.
Primary hyperoxaluria type 1 is a very rare genetic disease characterized by oxalate overproduction in the liver. Oxlumo is an RNA interference drug that targets a protein that encodes glycolate oxidase, which inhibits oxalate production.
The expanded FDA approval is based on six-month results from a phase 3 study in which Oxlumo demonstrated substantial reductions in plasma oxalate levels.