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news.FDA has approved Carbaglu (carglumic acid) tablets to treat a condition known as hyperammonemia caused by N-acetylglutamate synthase (NAGS) deficiency that results in elevation of ammonia levels in the blood.
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Reportedly, the safety and efficacy of Carbaglu was studied in 23 patients with NAGS who received the treatment for times ranging from six months to 21 years. In these patients, Carbaglu reduced blood ammonia levels within 24 hours and normalised ammonia levels within three days. The majority of those in the study appeared to maintain normal plasma ammonia levels with long-term Carbaglu treatment.
FDA said that Carbaglu should only be administered by a physician experienced in treating metabolic disorders. The recommended initial dose of Carbaglu is 100 to 250mg/kg/day for treatment of acute hyperammonemia. Use of other ammonia-lowering therapies with Carbaglu during episodes of acute hyperammonemia is recommended. Dosing should be adjusted based on a patient’s ammonia levels and symptoms.
Janet Woodcock, director of center for drug evaluation and research at FDA, said: “We are very excited that more drugs are being developed to treat very rare but often devastating genetic disorders. We hope to see continuing progress in this area.”