FDA Approves Wellstat’s Orphan Drug Xuriden

The FDA Sept. 4 approved Xuriden (uridine triacetate), the first treatment approved by the agency for patients with hereditary orotic aciduria.

Xuriden is marketed by Wellstat Therapeutics Corp., based in Gaithersburg, Md.

Hereditary orotic aciduria is a rare metabolic disorder, which has been reported in approximately 20 patients worldwide, the Food and Drug Administration said. Hereditary orotic aciduria is inherited from a recessive gene. The disease is due to a defective or deficient enzyme, which results in the body being unable to normally synthesize uridine, a necessary component of ribonucleic acid (RNA).

Signs and symptoms of the disease include blood ...