FDA approves Oxlumo, first drug to treat rare metabolic disorder

The US Food and Drug Administration has approved Oxlumo (lumasiran) as the first treatment for primary hyperoxaluria type 1 (PH1), a rare genetic disorder.

This approval is a cumulation of the work of experts and community members coordinated by the Oxalosis & Hyperoxaluria Foundation and the Kidney Health Initiative.

The FDA granted the approval of Oxlumo to RNAi therapeutics company Alnylam Pharmaceutical (Nasdaq: ALNY), just days after clearance by the European Medicines Agency. The FDA has granted a Priority Review for the NDA and set an action date of December 3, 2020 under the Prescription Drug User Fee Act.