Alnylam Pharma NDA for Oxlumo Approved

FDA has approved an Alnylam Pharmaceuticals NDA for Oxlumo (lumasiran) as the first treatment for primary hyperoxaluria Type 1 (PH1), a rare genetic disorder. Primary hyperoxalurias (PHs) are caused by excess production of oxalate, a substance consumed in food and also produced by the body, an FDA release says. “PH1 affects an estimated one to three individuals per million in North America and Europe and accounts for approximately 80% of PH cases,” it says.

Oxlumo works to decrease oxalate production. Approval was based on data from two studies in patients with PH1. In the first study, patients in Oxlumo group had a 65% reduction of oxalate in the urine, compared to an average 12% reduction in the placebo group, the agency says. In the second study, oxalate patients had a 71% decrease in urinary oxalate by the sixth month of the study. The most common side effects of Oxlumo include injection site reaction and abdominal pain.