Carbaglu Approved for Rare Genetic Disorder

THURSDAY March 18, 2010 -- 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.

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.

Posted: March 2010


View comments

Hide
(web4)