SILVER SPRING, Md. The Food and Drug Administration has approved a drug for treating a condition that causes too much ammonia in the blood.
The FDA announced Thursday the approval of Carbaglu (carglumic acid) tablets, made by Orphan Europe for treating N-acetylglutamate synthase deficiency, an extremely rare genetic disorder that can show up in babies soon after birth. The disease, also known as NAGS deficiency, can be fatal if not detected and treated rapidly.
“We are very excited that more drugs are being developed to treat very rare but often devastating genetic disorders,” FDA Center for Drug Evaluation and Research director Janet Woodcock said. “We hope to see continuing progress in this area.”
In clinical trials, Carbaglu was shown to reduce blood ammonia levels within 24 hours and bring them to normal within three days.