The Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) announced on December 16, 2024 that VGR-R01, a gene therapy developed by Shanghai Vitalgen BioPharma Co., Ltd. (hereinafter referred to as “Vitalgen”) for treating Bietti Crystalline Dystrophy (BCD) has been incorporated in the pilot project of Patient-Centered Drug Research and Development Pilot Work Plan for Rare Diseases (“Care Plan”).
The VGR-R01 project is currently in the enrollment stage of a Phase III clinical study. Vitalgen explored the changes in visual function of BCD patients after subretinal injection of VGR-R01 in different clinical outcome evaluations through Phase I/II clinical studies, and validated the superior efficacy and safety of the product in BCD patients.
The “Care Plan” was developed by the CDE to encourage and guide drug development organizations to listen to patients and learn about their experiences and feelings during the drug development, and to push patient-centered drug development and marketing for rare diseases. It came into effect on September 13, 2024. The plan divides drug development into 5 stages, and the CDE selects 1~2 representative varieties for inclusion in the “Care Plan” at each stage. The inclusion of VGR-R01 in the “Care Plan” represents the CDE’s recognition of VGR-R01 innovation and patient benefits. Vitalgen will subsequently conduct Phase III clinical studies in accordance with the implementation framework and report to the CDE on the progress or results of the work required in the implementation framework, to jointly advance the drug development and marketing of VGR-R01 in the field of rare diseases of BCD.
About VGR-R01
VGR-R01 is a gene therapy product developed by Vitalgen for recombinant adeno-associated viruses. This product, based on the strategy of gene replacement therapy, uses AAV8 coat protein to mediate the transduction of RPE cells and transduce the CYP4V2 gene expression cassette carrying CYP4V2 gene into the nucleus of the cell, while expressing the CYP4V2 protein and thus correcting the fatty acid metabolism disorder in the patient's retina, for clinical results of preventing or improving the structural and/or functional damage of RPE cells and photoreceptor cells and choroid, correcting visual impairment, protecting residual visual function, or delaying vision deterioration.
The product was approved by the NMPA to conduct clinical trials for the treatment of BCD in November 2022, making it the world's first gene therapy drug to enter the registered clinical trials stage in this treatment field. The Phase III clinical trial is currently in the recruitment phase and has started 6 study sites, namely, Beijing Tongren hospital affiliated to Capital Medical University, Shanghai First People's Hospital, the First Hospital Affiliated to the Army Medical University, Zhongshan Ophthalmic Center of Sun Yat-sen University, Affiliated Optometry Hospital of Wenzhou Medical University, and Eye, Ear, Nose and Throat Hospital of Fudan University.
Previously in August 2024, VGR-R01 for BCD was approved by the CDE for inclusion in the breakthrough therapeutic drug program based on clinically significant data on vital clinical outcomes. And in October of the same year, VGR-R01 was granted Orphan Drug Designation (ODD) by the US Food and Drug Administration.