Carbaglu Approved for Rare Genetic Disorder

Causes elevated ammonia in blood

Related Encyclopedia • Acidophilus • Antioxidants • Caffeine (and its effects) • Calcinosis More... Related Healthscout Videos Holiday Foods: Eat This, Not That Shrinking Stomachs -- A New Way A Cure for Diabetes? Medicine's Next Big Thing... Do Pills Equal Power? More... Related Drug Information • Klor-Con • Klor-Con ER More... Related News Articles • New Sweetness Detectors Found in Human Taste Cells • Eating Disorders More Prevalent Than Thought Among American Teens • Cartoon Branding Affects Kids' Cereal Choice, Regardless of Taste • Potassium-Rich Foods May Cut Stroke, Heart Disease Risk More...

THURSDAY, March 18 (HealthDay News) -- The drug Carbaglu (carglumic acid) has been approved by the U.S. Food and Drug Administration to treat a rare genetic disorder called N-acetylglutamate synthase (NAGS deficiency), which results in elevated blood levels of ammonia, the agency said Thursday.

The drug's safety and effectiveness were evaluated in 23 people with NAGS. Carbaglu reduced blood ammonia within a day and lowered levels to a normal range within three days, the FDA said.

Text Continues Below

---

The most common side effects included vomiting, abdominal pain, fever, inflamed tonsils, anemia, ear infection, diarrhea, nasal and throat inflammation, and headache.

The drug should only be administered by a doctor with experience in treating metabolic problems, the agency said. The medication is manufactured by Orphan Europe.

More information

To learn more about NAGS deficiency, visit Orphan Europe.

-- Scott Roberts