FDA Approves First Treatment for Hereditary Orotic Aciduria

Article

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.

Related Videos
Image Credit: SciePro - stock.adobe.com
Pharmacist selling medications in the pharmacy | Image Credit: rh2010 - stock.adobe.com
Atopic dermatitis on a patient's hand -- Image credit: Ольга Тернавская | stock.adobe.com
biosimilar word or concept represented by wooden letter tiles on a wooden table with glasses and a book | Image Credit: lexiconimages - stock.adobe.com
Image credit: alicja neumiler | stock.adobe.com
Laboratory test tubes and solution with stethoscope background | Image Credit: Shutter2U - stock.adobe.com
Laboratory test tubes and solution with stethoscope background | Image Credit: Shutter2U - stock.adobe.com
Image credit: Krakenimages.com | stock.adobe.com
Human brain digital illustration. Electrical activity, flashes, and lightning on a blue background. | Image Credit: Siarhei - stock.adobe.com
© 2024 MJH Life Sciences

All rights reserved.