Posted by Michael Wonder on 24 Nov 2020
FDA approves first drug to treat rare metabolic disorder
23 November 2020 - Today, the U.S. FDA approved Oxlumo (lumasiran) as the first treatment for primary hyperoxaluria type 1, 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.
Oxlumo received orphan drug designation, which provides incentives to assist and encourage drug development for rare diseases. The application was also granted breakthrough therapy designation. In addition, the manufacturer received a rare pediatric disease priority review voucher.
