WuXi Biologics and CANbridge Celebrate Approval of Innovative Therapy for Gaucher Disease in China
Saturday, May 17, 2025
WuXi Biologics has announced the approval of velaglucerase-beta for injection (Gaurunning), developed in partnership with CANbridge Pharmaceuticals, by China’s National Medical Products Administration (NMPA).
This marks the first and only domestically developed enzyme replacement therapy (ERT) approved in China for the treatment of Gaucher disease in adolescents aged 12 and above, and adults with Type I and Type III forms of the condition.
Gaucher disease is a rare inherited lysosomal storage disorder caused by a deficiency of the enzyme glucocerebrosidase. It leads to the build-up of harmful substances in certain cells and organs, resulting in symptoms such as enlarged liver and spleen, anaemia, bone pain, and neurological complications. The global prevalence of Gaucher disease ranges from 0.7 to 1.75 per 100,000 people, and it is included in China’s official list of rare diseases.
Velaglucerase-beta for injection represents the first rare disease drug collaboration between CANbridge and WuXi Biologics. The project utilised WuXi Biologics’ integrated technology platform to move from early development to commercial production. It involved the use of advanced cell line technology and the WuXiUP™ continuous bioprocessing system, which led to over a 110-fold increase in yield and a more than 50% rise in enzyme activity. These improvements have contributed to the therapy’s affordability and quality assurance. Furthermore, the therapy is the first biological product in China to pass segmented manufacturing inspection.
Gaucher disease is caused by autosomal recessive mutations in the GBA gene and affects both males and females. It includes several types, with Types I and III being the most common forms that allow survival into adulthood. The disease leads to a range of complications, including blood disorders and skeletal issues, with more severe types causing early neurological decline. ERT has been the global standard of care for over 30 years, consistently improving non-neurological symptoms and quality of life.
The development of velaglucerase-beta included the creation of specialised cell-based assays to support product release and stability testing, ensuring high standards of quality control. The product has now become the first commercialised in-house rare disease therapy from CANbridge, following the earlier launches of Hunterase® and Livmarli®.
WuXi Biologics continues to support the global development of rare disease therapies through its CRDMO model and currently hosts 21 rare disease projects on its platform. The company also promotes public awareness through campaigns such as "Run for Health", which has engaged thousands of participants, and educational programmes offering scholarships to students living with rare diseases in China.
This recent approval underscores the importance of collaboration in accelerating drug development and improving access for patients with rare conditions.
Source: prnewswire.com
