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VGM-R02b Obtained Rare Pediatric Disease Designation (RPDD) from FDA

2022-05-25

On May 25, 2022 (Eastern Standard Time), Shanghai Vitalgen BioPharma Co., Ltd. was awarded Rare Pediatric Disease Designation (RPDD) for the drug candidate VGM-R02b in treating glutaric aciduria type I (GA-1) by the Food and Drug Administration (FDA) . This is the first gene therapy product for GA-I worldwide. The designation will qualify Vitalgen upon approval of VGM-R02b for an assignable and transferable priority review voucher (PRV) that can be used to speed up the approval process for any other drug.



ABOUT GA-1:
GA-I is a rare inherited metabolic disease, cause by GCDH (glutaryl-CoA dehydrogenase) gene mutation. The deficiency of GCDH leads to the metabolic disorder of L-lysine, L-hydroxylysine and L-tryptophan, which results in an accumulation of GA (glutaric acid) and 3-OH-GA (3-hydroxyglutaric acid) in body tissues, causing multiple organ dysfunction in particular in the CNS. Acute encephalopathic crises usually occurs in infants and young children that are triggered by infectious diseases, high protein diet, vaccinations, which is life-threatening. VGM-R02b is a potential treatment to protect infants and children from severe or life-threatening disease caused by glutaric acidemia.

ABOUT RPDD AND PRV:
RPDD is an important incentive measure provided by FDA to facilitate the development of pediatric rare disease products. The PRV qualifies the sponsor at the time of registration of RPDD product for a salable, transferable priority review voucher which can be used to speed up the approval process for another drug candidate.