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FDA Approves Drug to Treat Hyperammonemia ROCKVILLE, Md -- March 18, 2010 -- The US Food and Drug Administration (FDA) has approved carglumic acid tablets (Carbaglu) to treat hyperammonemia caused by N-acetylglutamate synthase (NAGS) deficiency -- an extremely rare, genetic disorder that can be present in babies soon after birth. NAGS deficiency which results in hyperammonemia can be fatal if it is not detected and treated rapidly. DNA testing can confirm the diagnosis of NAGS. “We are very excited that more drugs are being developed to treat very rare but often devastating genetic disorders” said Janet Woodcock, MD, FDA’s Center for Drug Evaluation and Research, Rockville, Maryland. “We hope to see continuing progress in this area.” The safety and efficacy of carglumic acid was studied in 23 patients with NAGS who received the treatment for times ranging from 6 months to 21 years. In these patients, carglumic acid reduced blood ammonia levels within 24 hours and normalised ammonia levels within 3 days. The majority of those in the study appeared to maintain normal plasma ammonia levels with long-term treatment. Side effects included vomiting, abdominal pain, fever, tonsillitis, anaemia, ear infection, diarrhoea, inflammation of the nose and throat, and headache. As with all FDA-approved products, the agency will continue to monitor carglumic acid as it is used to treat hyperammonemia. Carglumic acid should only be administered by a physician experienced in treating metabolic disorders. The recommended initial dose is 100 to 250 mg/kg/day for treatment of acute hyperammonemia. Use of other ammonia-lowering therapies with carglumic acid during episodes of acute hyperammonemia is recommended. Dosing should be adjusted based on a patient’s ammonia levels and symptoms. SOURCE: US Food and Drug Administration E-mail this page to a friend or colleague! To print, use this version