FDA Approves Xuriden to Treat Patients with Rare Metabolic Disorder

FDA Approves Xuriden to Treat Patients with Rare Metabolic Disorder
September 14, 2015

Last week, the U.S. Food and Drug Administration (FDA) issued its first approval of a treatment for hereditary orotic aciduria. The drug is called Xuriden (uridine triacetate) and was developed by the American biopharmaceutical company Wellstat Therapeutics Corporation.

Hereditary orotic aciduria is a rare metabolic disorder, which has been reported in approximately 20 patients worldwide. The condition 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 abnormalities, urinary tract, failure to thrive, and developmental delays.

"Today's approval and rare pediatric disease priority review voucher underscore the FDA's commitment to making treatments available to patients with rare diseases," Amy G. Egan, deputy director of the Office of Drug Evaluation III in the FDA's Center for Drug Evaluation and Research (CDER), said in a written statement. "Prior to Xuriden's approval, patients with this rare disorder had no approved treatment options."

Xuriden has been granted a designation as an "orphan drug" because it treats a rare disease. This designation provides financial incentives, like clinical trial tax credits, user fee waivers, and eligibility for market exclusivity. The drug also received a priority review, which allows for an expedited introduction into the marketplace.

The safety and effectiveness of Xuriden were evaluated in a single arm, six-week, open-label trial in four patients with hereditary orotic aciduria, ranging in age from three to 19 years of age, and in a six-month extension phase of the trial. At both the six-week and six-month assessments, Xuriden treatment resulted in stability of the hematologic parameters in all four clinical trial patients. The safety and effectiveness of uridine replacement therapy were further supported by case reports from the published literature.

According to BusinessWire, Wellstat believes that Xuriden will be commercially available in early 2016.