First Drug for Hereditary Orotic Aciduria Approved

The Food and Drug Administration (FDA) has approved Xuriden (uridine triacetate; Wellstat Therapeutics) oral granules for the treatment of hereditary orotic aciduria, the first approved treatment for this rare metabolic disorder.

The FDA approval was based on a six-week, single arm, open-label trial (n=4) in patients ranging in age from 3–19 years, as well as a six-month extension phase of the trial. The study evaluated changes in the patients' pre-specified hematologic parameters during the study duration. Treatment with Xuriden led to stability of hematologic parameters in all study patients at both the six-week and 6-month assessments. No side effects were reported in patients treated with Xuriden for up to nine months.

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Xuriden is a pyrimidine analog for uridine replacement that provides 4–7 times more uridine than oral uridine administration itself. The oral granules can be mixed with food or in milk or infant formula. Uridine replacement therapy provides the uridine that patients cannot make adequately themselves in order to restore intracellular uridine nucleotide concentrations. This allows and supports the biosynthetic functions of pyrimidine nucleotides and their derivatives, and also normalizes orotic acid production.

Xuriden was designated as an orphan drug and has been granted priority review by the FDA. Wellstat Therapeutics was also granted a rare pediatric disease priority review voucher. Xuriden will be available as orange-flavored granules (95% w/w) in single-use 2g packets in 30-count cartons. It is expected to launch in early 2016.

For more information call (800) 914-0071 or visit WellstatTherapeutics.com.

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