FDA Approves Wellstat’s Rare Metabolic Disorder Drug

The FDA earlier this month approved Wellstat Therapeutics’s Xuriden to treat a rare metabolic disorder known as hereditary orotic aciduria, landing the company a rare pediatric disease priority review voucher.

Approval of Xuriden (uridine triacetate) was based on data showing stability of prespecified hematologic parameters in all four patients in a clinical trial.

Prior to Xuriden’s approval, patients with hereditary orotic aciduria had no approved treatment options, the FDA says. Xuriden was granted orphan drug designation since the rare disorder has been reported in only 20 patients worldwide.

Priority review vouchers can be used to get a six-month priority review of another NDA or BLA. They can also be sold, which was done recently when United Therapeutics sold one to AbbVie for $350 million.

Gaithersburg, Maryland-based Wellstat plans to launch the product next year, spokesman Jeremiah Hall said. — Jonathon Shacat