China NMPA Approves CANbridge’s Velaglucerase-Beta for Gaucher Disease

CANbridge Pharmaceuticals Inc., a global biopharmaceutical company, announced that velaglucerase-beta for injection (Gaurunning), with its wholly - owned subsidiary, CANbridge (Shanghai) Life Sciences Ltd. as the holder, has obtained marketing approval from the National Medical Products Administration (NMPA). Gaurunning is the first domestically independently developed long-term enzyme replacement therapy for adolescents aged 12 and above and adults with type I and type III Gaucher disease. As a Class 1 innovative drug, it can fully replace similar imported products and will significantly enhance medication accessibility for domestic patients.

In August of 2024, CANbridge announced positive top-line results from the pivotal clinical trial of Gaurunning in adult and pediatric Gaucher disease patients. In September, the Center for Drug Evaluation (CDE) of NMPA granted it priority review status. In October of the same year, CANbridge successfully passed the Manufacturing Authorization Holder (MAH) production and quality management system audit and on-site inspection and obtained the Drug Manufacturing License Type B certificate. In November, the New Drug Application (NDA) for Gaurunning was accepted by NMPA. In March 2025, it successfully passed the Pre-approval inspection and pre-marketing GMP compliance inspection for the pilot biological product of Divided manufacturing. Velaglucerase-beta for injection is the first innovative biological product in China to pass the inspection of Divided manufacturing of biological products.

James Xue, Ph.D., CANbridge founder, chairman, and CEO, stated: ""Enzyme replacement therapy (ERT) is a critical component of CANbridge's global pipeline strategy. 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, as well as the professionalism in pharmaceutical regulatory affairs but also demonstrates the recognition and support of China's drug regulatory authorities for the urgent medical needs of Gaucher disease patients.

The innovative technology adopted in Gaurunning is enzyme modification and directed evolution, serving as a typical case of new quality productive forces. 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. Going forward, the platform-based technology of Gaurunning will significantly reduce development costs, enabling patients to access safe, effective, and affordable domestic enzyme replacement therapies. The successful development and approval of Gaurunning embody the original entrepreneurial aspiration of CANbridge. It owes much to the concerted efforts of numerous partners including WuXi Biologics, benefits from the support of national and local regulatory authorities, relevant departments, and medical institutions. Above all, it carries the hope that patients with Gaucher disease across China will soon gain access to sustainable treatment.

Gaurunning is the first collaborative project between CANbridge and WuXi Biologics (2269.HK) in the rare disease field. We thank our partners, including WuXi Biologics, for enabling the development and production of this product. This is not only the third rare disease treatment launched by CANbridge’s Hunterase and Livmarli but also the first self-developed product to enter commercialization. We will continue to deepen our focus on localized R&D, production, launch, and accessibility of rare disease drugs to bring innovative treatment solutions to Chinese patients.""

Dr. Chris CHEN, chief executive officer of WuXi Biologics, said: ""We are extremely honoured to have empowered CANbridge to achieve this key milestone of Gaurunning, which also represents a significant breakthrough in China's rare disease drug innovation. The journey of rare disease drug development is long and arduous, requiring the overcoming of countless technical hurdles and challenges from early laboratory discovery to ultimately benefiting patients. The successful realization of this product from concept to commercial production not only testifies to CANbridge's decade-long commitment to deepening its presence in the rare disease field but also serves as a successful application of WuXi Biologics' integrated technology platform and CRDMO business model. The shared vision of both parties in addressing the unmet clinical needs of rare disease patients has led to the achievement of China's first domestically developed enzyme replacement therapy for Gaucher disease. We look forward to empowering more global partners to accelerate the development of high-quality rare disease treatments, driving rare disease drug innovation, and benefiting more rare disease patients.""

Velaglucerase-beta for injection is the first domestically developed recombinant human glucocerebrosidase ERT in China, now approved for clinical use in treatment of Type I and Type III Gaucher disease patients. Most Gaucher disease patients are Type I and Type III, which are chronic non-neuronopathic and chronic neuronopathic forms, respectively. velaglucerase-beta is administered as an intravenous infusion and is intended to supplement the lack of glucocerebrosidase in the lysosomes of GD patients.  

The pivotal clinical trial of velaglucerase-beta achieved positive top-line data in August 2024. The velaglucerase-beta pivotal trial is a randomized, double-blind, dose-comparison study designed to evaluate the efficacy, safety, and pharmacokinetics of intravenous velaglucerase-beta administered every other week in newly treated Gaucher Disease (GD) patients, with an extension period. The results demonstrate 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).  The primary endpoint of this trial protocol has been agreed upon by the CDE.

Gaucher disease, one of the most common lysosomal storage disorders, 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. Gaucher disease is a clinical spectrum that comprises Type 0 (perinatal-lethal), Type I (chronic non-neuronopathic), Type II (acute neuronopathic), and Type III (chronic neuronopathic) forms, with Types I and III surviving into adulthood. Gaucher disease is caused by a deficiency of glucocerebrosidase (acid b-glucosidase), an enzyme that helps break down a cellular membrane glycosphingolipid called glucocerebroside (glucosylceramide) inside lysosomes. As a result, glucocerebroside accumulates primarily in cells of the monocyte-macrophage lineage (Gaucher cells) within certain organs, leading to splenomegaly, hepatomegaly, anaemia, thrombocytopenia, bone pain and fractures, and in the most severe forms (perinatal-lethal, Types II and III), early neurological symptoms.  For 30 years, recombinant human glucocerebrosidase enzyme replacement therapy (ERT) has been the standard of care for Gaucher disease, with clinical trials and real-world data demonstrating significant improvement in the major non-neurological signs and symptoms of disease and quality of life. There were 3,000 patients with Gaucher disease in China in 2020, according to Frost & Sullivan.

CANbridge Pharmaceuticals Inc. is a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare disease.

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