Clinical trial for Vitalgen's self-developed VGM-R02b accepted

On April 27, 2023, Shanghai Vitalgen BioPharma Co., Ltd. received the notification of clinical trial acceptance from the National Medical Products Administration for its self-developed VGM-R02b. VGM-R02b is used for the treatment of glutaric acidemia type I (GA-I) and is an innovative drug for children. It was previously granted Rare Pediatric Disease Designation (RPDD) status by the U.S. Food and Drug Administration (FDA) for the treatment of glutaric acidemia type I (GA-I).


Glutaric Acidemia Type I (GA-I), also known as Glutaric Aciduria Type I, is a rare autosomal recessive neurometabolic disorder that is included in China's First List of Rare Diseases. In GA-I patients, the decrease or defect of GCDH activity caused by mutations in the gene encoding glutaryl-coA dehydrogenase (GCDH) leads to metabolic disorders of lysine, hydroxylysine and tryptophan, and abnormal accumulation of metabolites glutaric acid and 3-hydroxyglutaric acid in the body, resulting in damage to multiple organs, mainly in the nervous system. It mostly develops in infancy and early childhood and often induces life-threatening acute encephalopathic crisis upon stress stimulation by infection, high protein intake or vaccination. VGM-R02b is a potential treatment option to prevent the progression of severe or life-threatening disease caused by glutaric acidemia in infancy and childhood.