Carbaglu receives FDA approval for a new indication to treat acute hyperammonemia associated with propionic acidemia and methylmalonic acidemia.-Recordati

Recordati, through its US subsidiary Recordati Rare Diseases Inc., announced the FDA has approved a new indication for Carbaglu (carglumic acid) tablets 200mg as adjunctive therapy to standard of care for the treatment of acute hyperammonemia due to propionic acidemia (PA) or methylmalonic acidemia (MMA) in pediatric and adult patients. Carbaglu is the first and only FDA approved medication for the treatment of acute hyperammonemia due to PA and MMA. Carbaglu was initially approved by the FDA for N-acetylglutamate synthase (NAGS) deficiency, another rare metabolic disorder, as adjunctive therapy to standard of care for the treatment of acute hyperammonemia due to NAGS deficiency, and maintenance therapy for the treatment of chronic hyperammonemia due to NAGS deficiency. There are few approved drugs that treat hyperammonemia, and none that are indicated for the treatment of acute hyperammonemia in PA (propionic acidemia) and MMA (methylmalonic acidemia ) patients”, said Mendel Tuchman, MD, Medical Geneticist and Professor Emeritus of Pediatrics at The George Washington University School of Medicine and Health Science. “Carbaglu has the potential to impact these patients by reducing high plasma ammonia levels during critical situations.”.