The Center for Drug Evaluation (CDE) website indicates that China-based Grand Pharmaceutical Group Limited’s (HKG: 0512) generic carglumic acid is set for priority review. The indications include hyperammonemia due to N-acetylglutamate synthase deficiency, hyperammonemia due to isovaleric acidemia, hyperammonemia due to methylmalonic acidemia, and hyperammonemia due to propionic acidemia.
Reason for Priority Review
The drug has been categorized as a “new pediatric variety, dosage form, and specification in line with children’s physiological characteristics,” which qualifies it for priority review.
Drug Background
Carglumic acid, originated by Italy-based Recordati, is a functional analog of N-acetylglutamate (NAG) and an activator of carbamoyl phosphate synthase 1 (CPS1). It is used to treat acute and chronic hyperammonemia associated with N-acetylglutamate synthase (NAGS) deficiency. The drug was first approved in the EU in January 2003 but never reached the China market. Grand Pharmaceutical filed its version for marketing approval in March of this year and was awarded priority review status, making it the second company to seek approval for the drug in China after Wuhan Wuyao Pharmaceutical.-Fineline Info & Tech
