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FDA Grants New Approval for Recordati’s Hyperammonemia Drug

January 28, 2021

Recordati Rare Diseases has received FDA approval for a new indication for its 200-mg Carbaglu (carglumic acid) tablets, allowing its use in treating acute hyperammonemia due to propionic acidemia or methylmalonic acidemia in pediatric and adult patients.

The company said the agency’s greenlight makes Carbaglu the first and only drug approved by the FDA for treating the rare inherited metabolic disorders that cause an excess of toxic metabolites.

The FDA first approved Carbaglu as an adjunctive therapy to standard of care for treating acute hyperammonemia due to another rare metabolic disorder, N-acetylglutamate synthase (NAGS) deficiency, and as a maintenance therapy for treating chronic hyperammonemia due to NAGS deficiency.

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