The FDA said the disorder, caused by a deficiency of the chemical N-acetylglutamate synthase, is an extremely rare genetic illness that can be present in babies soon after birth and can be diagnosed by DNA testing. The elevated levels of ammonia, a condition called hyperammonemia, can be fatal if it is not detected and treated rapidly.
"We are very excited that more drugs are being developed to treat very rare, but often devastating, genetic disorders" said Dr. Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research.
The FDA said Carbaglu (carglumic acid) reduced blood ammonia levels within 24 hours and normalized ammonia levels within three days during a clinical trial.
Carbaglu is manufactured by the French pharmaceutical company Orphan Europe.
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