By Fineline Cube 
China-based Yifan Pharmaceutical Co., Ltd (SHE: 002019) announced that the National Medical Products Administration (NMPA) has accepted for review its generic version of glycerol phenylbutyrate. This marks a significant step toward providing a new treatment option for patients with urea cycle disorders (UCDs).
Indications and Mechanism
Glycerol phenylbutyrate is intended for patients with UCDs who cannot achieve disease control through protein intake limitation and/or amino acid supplementation. These disorders include carbamoyl phosphate synthetase I deficiency, ornithine carbamyltransferase deficiency, Citrullinemia type 1, argininosuccinuria, argininemia, and hyperornithinemia hyperammonemia homocitrullinemia (HHH) syndrome. The oral formulation helps maintain normal blood ammonia levels, reduce high ammonia crises, and improve cognitive function and growth.
Market Context
Currently, in mainland China, Immedica Pharma AB is the sole provider of oral glycerol phenylbutyrate. In December 2020, WinHealth secured exclusive commercialization rights to the drug in Greater China and several Asia-Pacific regions through a licensing deal with Immedica Pharma AB. Yifan Pharmaceutical’s oral glycerol phenylbutyrate is poised to compete for the first generic approval, potentially expanding treatment access for patients with UCDs.-Fineline Info & Tech