FDA OKs Wellstat’s Orphan Drug
The US Food and Drug Administration has approved Wellstat Therapeutics Corporation’s Xuriden (uridine triacetate), for treating hereditary orotic aciduria, a rare metabolic disorder, which has been reported in approximately 20 patients worldwide. Wellstat is a biopharmaceutical company based in Gaithersburg, Maryland.
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 abnormalities (anemia, decreased white blood cell count, decreased neutrophil count), urinary tract obstruction due to the formation of orotic acid crystals in the urinary tract, failure to thrive, and developmental delays.
Xuriden is an orally administered product intended to replace uridine. Xuriden is approved as oral granules that can be mixed with food or in milk or infant formula, and is administered once daily.
The FDA granted Xuriden orphan drug designation because it treats a rare disease. Orphan drug designation provides financial incentives, such as clinical trial tax credits, user fee waivers, and eligibility for market exclusivity to promote rare disease drug development. Xuriden was also granted priority review. An FDA priority review provides for an expedited review of drugs for serious diseases or conditions that may offer major advances in treatment. The manufacturer of Xuriden was granted a rare pediatric disease priority review voucher, a provision that encourages development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.
In September 2014, Wellstat entered into an agreement with AstraZeneca under which the Pediatric Priority Review Voucher would be transferred to AstraZeneca upon approval. Financial terms of the agreement were disclosed.
Wellstat anticipates that Xuriden will be commercially available in early 2016.