CANbridge Pharmaceuticals Inc. has achieved a significant milestone in rare disease treatment with the approval of velaglucerase-beta for injection (Gaurunning) by China's National Medical Products Administration (NMPA). Announced on May 16, 2025, this marks the first domestically developed enzyme replacement therapy (ERT) for Gaucher disease in China.
The approval specifically covers adolescents aged 12 and above and adults with Type I and Type III Gaucher disease. As a Class 1 innovative drug, Gaurunning is positioned to replace imported alternatives and significantly enhance medication accessibility for Chinese patients.
"Enzyme replacement therapy is a critical component of CANbridge's global pipeline strategy," said Dr. James Xue, Founder, Chairman, and CEO of CANbridge Pharmaceuticals. "The approval of Gaurunning in China is a milestone. The six-month timeframe from NDA acceptance to approval not only reflects the strong safety, effectiveness and reliability of our product, but also demonstrates the recognition and support of China's drug regulatory authorities for the urgent medical needs of Gaucher disease patients."
Accelerated Regulatory Pathway
The development of Gaurunning followed an expedited regulatory timeline. In August 2024, CANbridge announced positive top-line results from the pivotal clinical trial. The Center for Drug Evaluation (CDE) of NMPA granted priority review status in September 2024, and by November, the New Drug Application was accepted. The drug successfully passed pre-approval inspection and pre-marketing GMP compliance inspection in March 2025, becoming the first innovative biological product in China to pass the inspection of divided manufacturing of biological products.
Clinical Efficacy and Trial Design
The pivotal clinical trial for velaglucerase-beta was a randomized, double-blind, dose-comparison study evaluating the efficacy, safety, and pharmacokinetics of intravenous administration every other week in newly treated Gaucher Disease patients.
Results demonstrated that the study successfully met its primary efficacy endpoint, showing a statistically significant mean percentage reduction from baseline in spleen volume at nine months for both the 60 U/kg dose (P<0.0001) and the lower 30 U/kg dose (P<0.001).
Technological Innovation
Gaurunning represents the first collaborative project between CANbridge and WuXi Biologics in the rare disease field. The development utilized innovative technology to construct high-expression cell lines and achieved significant improvements in both yield and quality through WuXi Biologics' WuXiUP™ platform.
"The project utilized innovative technology to construct high-expression cell line and achieved significant improvements in both yield and quality," noted Dr. Chris Chen, CEO of WuXi Biologics. "The overall yield increased by more than 110 times, and the specific enzyme activity increased by more than 50%, ensuring affordability for Gaucher disease patients."
The manufacturing process also incorporated cell-based bioassay methods for commercial product release and stability testing, addressing technical challenges of enzyme testing and ensuring product quality control.
Understanding Gaucher Disease
Gaucher disease is one of the most common lysosomal storage disorders, affecting an estimated 3,000 patients in China as of 2020, according to Frost & Sullivan. It is a rare inherited genetic metabolic disease caused by autosomal recessive mutations in the GBA gene located on chromosome 1q22 and affects both males and females equally.
The disease is characterized by a deficiency of glucocerebrosidase (acid β-glucosidase), an enzyme that helps break down a cellular membrane glycosphingolipid called glucocerebroside inside lysosomes. This deficiency leads to glucocerebroside accumulation primarily in cells of the monocyte-macrophage lineage (Gaucher cells) within certain organs.
Clinical manifestations include splenomegaly, hepatomegaly, anemia, thrombocytopenia, bone pain and fractures, and in the most severe forms, early neurological symptoms. Gaucher disease has been included in China's "First List of Rare Diseases."
Implications for Patient Access
For 30 years, recombinant human glucocerebrosidase enzyme replacement therapy has been the standard of care for Gaucher disease globally. Clinical trials and real-world data have demonstrated significant improvement in the major non-neurological signs and symptoms of disease and quality of life.
"The innovative technology adopted in Gaurunning is enzyme modification and directed evolution, serving as a typical case of new quality productive forces," Dr. Xue explained. "In the future, combined with continuous flow production process technology, it can be extended to other similar drugs beyond Gaucher disease to achieve large-scale, batch production."
The platform-based technology is expected to significantly reduce development costs, enabling patients to access safe, effective, and affordable domestic enzyme replacement therapies.
Expanding Rare Disease Portfolio
This approval represents CANbridge's third rare disease treatment following Hunterase and Livmarli, but notably is the company's first self-developed product to enter commercialization. CANbridge has committed to deepening its focus on localized R&D, production, launch, and accessibility of rare disease drugs to bring innovative treatment solutions to Chinese patients.
CANbridge Pharmaceuticals has a differentiated drug portfolio with now 3 approved drugs and a pipeline of 8 assets targeting prevalent rare disease indications with unmet needs. The company's Next-Generation Innovation and Process Development Facility is also developing novel, potentially curative gene therapies for rare genetic diseases, including Pompe disease, Fabry disease, spinal muscular atrophy, Duchenne muscular dystrophy, and other neuromuscular conditions.
The approval of Gaurunning represents a significant advancement in China's capabilities to develop innovative treatments for rare diseases domestically, potentially opening the door for more affordable and accessible therapies for patients with rare conditions throughout the country.
