Uridine triacetate approved to treat rare autosomal recessive disorder

U.S. Food and Drug Administration approved uridine triacetate, the first FDA-approved treatment for patients with hereditary orotic aciduria. The FDA granted Uridine triacetate orphan drug designation because it treats a rare disease.
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.
Uridine triacetate is an orally administered product intended to replace uridine. Uridine triacetate is approved as oral granules that can be mixed with food or in milk or infant formula, and is administered once daily.