Drug Information Update - FDA approves new orphan drug to treat rare
autosomal recessive disorder
FDA Division of Drug Information: Know the Moment It Happens

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On Friday September 4, 2015, the U.S. Food and Drug Administration
approved Xuriden (uridine triacetate), the first FDA-approved treatment
for patients with hereditary orotic aciduria. Hereditary orotic aciduria
is a rare metabolic disorder, which has been reported in approximately
20 patients worldwide.

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.

No side effects were observed in patients treated with Xuriden for up to
nine months.

For more information, please visit: Xuriden