Shanghai Vitalgen BioPharma Co., Ltd. (hereinafter referred to as "Vitalgen" or the "Company") recently announced that VGN-R09b, its independently developed gene therapy product for the treatment of Aromatic L-Amino Acid Decarboxylase Deficiency (AADCD), has been proposed for inclusion in the Breakthrough Therapy Designation (BTD) list by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA). VGN-R09b is the company's second gene therapy product, following VGR-R01, to receive Breakthrough Therapy Designation and enter Phase III clinical trials, further demonstrating the company's sustained innovation capabilities and pipeline depth in the field of gene therapy.
Following the granting of BTD, the company will maintain close communication with the drug evaluation authority regarding the product throughout the subsequent development process and may have opportunities to further accelerate its path to market through procedures such as Priority Review and/or Conditional Approval. The proposed inclusion of VGN-R09b in the Breakthrough Therapy Designation list signifies the CDE's recognition of the product's significant advantages in important clinical outcomes and its potential to address the current treatment gap for AADCD in China.
Dr. Zhao Xiaoping, CEO of the company, stated, "The successful proposed inclusion of VGN-R09b in the Breakthrough Therapy Designation list represents important recognition of its clinical value and the company's R&D direction. Having two consecutive products receive BTD fully demonstrates the forward-looking nature of our R&D strategy and the efficient execution capability of our team. We will actively leverage the policy support provided by this designation, engage in even closer communication with the CDE, and efficiently advance the clinical research and market approval process for VGN-R09b to fill the treatment gap in the AADCD field in China."
Currently, the Phase I/III registrational clinical study of VGN-R09b for AADCD is being conducted at Shanghai Children's Medical Center and Children's Hospital of Fudan University. Available data indicate that VGN-R09b is well-tolerated, with no serious adverse events related to the product or the surgical procedure observed. All 13 treated patients have demonstrated significant motor function improvement. Among these, 10 patients have achieved motor developmental milestones (the primary efficacy endpoint), with 5 patients achieving this endpoint as early as three months post-administration. The overall efficacy trend is significantly superior to currently marketed products for the same indication outside of China, demonstrating promising therapeutic potential.
About AADCD
AADCD is an autosomal recessive inherited neurometabolic disorder caused by pathogenic mutations in the DDC gene, leading to severe deficiencies in the synthesis of key neurotransmitters such as dopamine and serotonin. Affected infants present with severe motor dysfunction, autonomic nervous system disturbances, and intellectual developmental delay. Most fail to achieve motor developmental milestones such as head control, sitting independently, and walking. They face a high risk of mortality during infancy and early childhood, and their quality of life is severely impacted. Currently, there is no effective curative therapy for this disease in China. Conventional treatments can only partially alleviate symptoms and cannot repair the underlying neurological deficits, with particularly limited effects in severely affected patients.
About VGN-R09b
VGN-R09b utilizes a recombinant adeno-associated virus (rAAV) as a vector. Administered via a one-time intraparenchymal injection, it precisely delivers the therapeutic gene to the site of disease, enhancing dopamine synthesis in the brain while promoting neuroprotection, with the potential to achieve a fundamental treatment for the disease. VGN-R09b received approval from the National Medical Products Administration (NMPA) on April 19, 2024, to conduct clinical trials in China for Aromatic L-Amino Acid Decarboxylase Deficiency (AADCD) and Idiopathic Parkinson's Disease (PD), and the relevant registrational clinical studies are currently being actively advanced. Additionally, the product received approval from the U.S. Food and Drug Administration (FDA) on July 26, 2024, to conduct clinical studies for Idiopathic PD, and was granted Fast Track Designation (FTD) by the FDA on June 25, 2025.