1. Nov 25, 2020

FDA approves first treatment for primary hyperoxaluria type 1

The U.S. Food and Drug Administration has approved lumasiran (Oxlumo; Alnylam Pharmaceuticals, Inc) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients, according to a press release.

This is the first treatment for PH1, a rare genetic disorder characterized by oxalate overproduction.

In the phase 3 ILLUMINATE-A trial, lumasiran significantly reduced levels of urinary oxalate compared to placebo. In the ILLUMINATE-B pediatric phase 3 study, the safety and efficacy of lumasiran were shown in patients <6 years.

“The approval of Oxlumo represents a great triumph of community involvement to address a rare disease. It is a result of input from patients, treating physicians, experts and sponsors at a patient-focused drug development meeting and through other collaborative efforts,” said Norman Stockbridge, MD, PhD, director of the Division of Cardiology and Nephrology in the FDA’s Center for Drug Evaluation and Research, in a statement.

Read the full press release here.