FDA approves Xuriden (uridine triacetate) for hereditary orotic aciduria- Wellstat Therapeutics

The FDA has approved Xuriden (uridine triacetate), from Wellstat Therapeutics, the first FDA-approved treatment for patients with hereditary orotic aciduria. The FDA granted Xuriden orphan drug designation and it was also granted priority review.

The safety and effectiveness of uridine triacetate 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. The study assessed changes in the patients’ pre-specified hematologic parameters during the trial period. At both the six-week and six-month assessments, uridine triacetate treatment resulted in stability of haematologic 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. No side effects were observed in patients treated with uridine triacetate for up to nine months.