Today, Shanghai Vitalgen BioPharma Co., Ltd. (hereinafter referred to as the "Company") announced that VGN-R08b, its independently developed AAV gene therapy product for the treatment of Gaucher disease, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA).
This designation marks the third key FDA recognition for VGN-R08b in the field of Gaucher disease, underscoring the FDA's continued acknowledgment of the therapeutic potential of the Company's product in this indication. Previously, VGN-R08b had been granted Rare Pediatric Disease Designation (RPDD) in October 2022 and Fast Track Designation (FTD) in October 2025. Furthermore, VGN-R08b received Investigational New Drug (IND) approval from China's National Medical Products Administration (NMPA) in 2025, steadily advancing its global development footprint.
This ODD from the FDA brings substantial regulatory and commercial benefits for the subsequent development and potential commercialization of VGN-R08b in the United States. These benefits include seven years of market exclusivity upon approval, tax credits for qualified clinical research costs, and more frequent guidance from the FDA during the review process. Such support will significantly propel the global clinical development of this product, offering new hope for a potential curative therapy to patients with Gaucher disease in urgent need of effective treatment options, particularly children suffering from the severe neuronopathic forms of Gaucher disease (nGD).
Dr. Zhao Xiaoping, CEO of the company, stated, "The granting of ODD by the U.S. FDA for VGN-R08b is a significant validation of our scientific innovation and development strategy. From RPDD to FTD, and now ODD, this series of regulatory milestones provides sustained momentum for our subsequent global clinical development. We will accelerate our efforts to bring this potentially curative therapy to patients worldwide as soon as possible."
About Gaucher Disease (GD)
Gaucher disease (GD) is a rare, inherited lysosomal storage disorder caused by mutations in the GBA1 gene, leading to deficient activity of the enzyme glucocerebrosidase (GCase). This deficiency results in the accumulation of substrate in multiple organs, causing progressive hepatosplenomegaly, skeletal erosion, and neurological damage, among other severe consequences. Among these, patients with neuronopathic Gaucher disease (nGD, including types 2 and 3) often present in infancy or early childhood with rapidly progressing disease. Currently, there is a lack of effective treatment options worldwide, representing a significant unmet medical need.
About VGN-R08b
VGN-R08b is a recombinant AAV-based gene replacement therapy designed specifically for GBA1 gene mutations. It aims to restore GCase activity at its source by delivering a functional GBA1 gene, thereby ameliorating metabolic abnormalities and alleviating neurological damage. As the world's only investigational gene therapy for nGD and a first-in-China program, VGN-R08b has demonstrated a favorable safety profile and potential to slow disease progression in an ongoing investigator-initiated trial (VGN-R08b-001, NCT06272149) conducted at Xinhua Hospital affiliated to Shanghai Jiao Tong University School of Medicine. A registrational clinical study of VGN-R08b for nGD has been approved in China.