FDA Approves First Treatment for Hereditary Orotic Aciduria

The rare disease only afflicts 20 people worldwide.

The rare disease only afflicts 20 people worldwide.

The FDA this week approved Xuriden for the treatment of patients with hereditary orotic aciduria.

The medication is the first FDA-approved treatment for patients with the disease. Hereditary orotic aciduria is a rare metabolic disorder that affects only 20 people worldwide.

It is inherited from a recessive gene that results in a defective or deficient enzyme. The defective or deficient enzyme disallows the body to synthesize uridine, a necessary component of ribonucleic acid (RNA).

Signs and symptoms include blood abnormalities such as decreased white blood cell count, urinary tract obstruction due to the formation of orotic acid crystals in the urinary tract, failure to thrive, and developmental delays.

No side effects were reported from the use of Xuriden in patients with the disease for up to 9 months post treatment.