China-based Grand Pharmaceutical Group Limited (HKG: 0512) has announced that it has received marketing approval from the National Medical Products Administration (NMPA) for its generic version of Italy’s Recordati’s Carbaglu (carglumic acid). This marks the first approval of such a generic drug in China, offering a treatment for hyperammoniaemia caused by N-acetylglutamate synthetase (NAGS) deficiency, isovaleric acidemia (IVA), methylmalonic acidemia (MMA), or propionate acidemia (PA).
Understanding Congenital Hyperammonemia and Its Impact
Congenital hyperammonemia is a rare disease resulting from defects in metabolic pathway-specific enzymes beyond the urea cycle. It is characterized by abnormally high blood ammonia levels and central nervous system dysfunction. Hyperammoniaemia is a critical condition in clinical practice, with excessive blood ammonia concentration being highly toxic to the nervous system. This toxicity can affect energy metabolism, inhibit nerve conduction, and lead to neurological dysfunction, causing irreversible brain damage.
Carglumic Acid’s Mechanism and Clinical Benefits
Carglumic acid is a functional analogue of N-acetylglutamate (NAG) and an activator of carbamoyl phosphate synthase 1 (CPS1). It can bind with unsynthesized ammonium bicarbonate to generate NAG, thereby stimulating the urea cycle. This process helps convert ammonia into urea, reducing blood ammonia concentration and improving or preventing brain damage caused by hyperammonemia. The drug was first approved in the European Union in January 2003 and received approval in China in June this year.
Competition and Regulatory Status
Meanwhile, Jiedi Pharma’s generic version of Carbaglu is awaiting regulatory decisions, having been granted priority review status in May 2023. The approval of Grand Pharmaceutical’s generic Carbaglu is a significant step forward in providing treatment options for patients with hyperammonemia in China.-Fineline Info & Tech
