CANbridge Pharmaceuticals CAN103 Investigational New Drug Application Approved in China for Treatment of Gaucher Disease


Beijing, China, Cambridge, Massachusetts, October 20, 2021 – CANbridge Pharmaceuticals Inc., a leading China-based global rare disease-focused biopharmaceutical company committed to the research, development and commercialization of transformative therapies, announced that the Investigational New Drug (IND) application for CAN103 has been approved by the Chinese National Medical Products Administration (NMPA). CAN103 is an enzyme replacement therapy (ERT) being developed by CANbridge as part of its rare disease partnership with WuXi Biologics (2269.HK) for the long-term treatment of adults and children with Gaucher disease, Types I and III.

“This approval, in a record 52 working days of CDE review, is a major win for Chinese Gaucher patients,” said James Xue, Ph.D. Founder, Chairman and CEO of CANbridge Pharmaceuticals Inc. “ We are working diligently to  commence our clinical trial program in CAN103, one of the candidates from our partnership with WuXi Biologics.Despite the fact that Gaucher disease treatments have been available in China for decades, most patients do not have access, underscoring the need for China-based treatments and solutions.”


About Gaucher disease (GD)

Gaucher disease is one of the most common lysosomal storage disorders (LSs). It is a rare hereditary genetic metabolic disease caused by a recessive mutation in the GBA gene, located on chromosome 1, and affects both males and females. It causes a deficiency of glucocerebrosidase, an enzyme that helps break down the muscle cell membrane component, glucocerebroside. As a result, glucocerebroside accumulates in cells and certain organs, a process which is characterized by splenomegaly, hepatomegaly, anemia, thrombocytopenia, bone pain and neurological symptoms. For nearly 30 years, glucocosylase ERT has been the standard treatment for Gaucher disease, with clinical trial and real-world data demonstrating significant improvement in the major non-neurological symptoms and quality of life. There were 3,000 patients with Gaucher disease in China in 2020, according to Frost & Sullivan.


About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. is a China-based global rare disease-focused biopharmaceutical company committed to the research, development and commercialization of transformative therapies.

CANbridge has a comprehensive and differentiated pipeline of 13 drug assets with significant market potential, targeting some of the most prevalent rare diseases and rare oncology.

These include Hunter syndrome (MPS II) and other lysosomal storage disorders (LSDs), complement mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases as well as glioblastoma multiforme (GBM).

CANbridge strategically combines global collaborations and internal research to build and diversify its drug portfolio and invest in next-generation gene therapy technologies for rare disease treatments. CANbridge global partners include, but are not limited to, Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the University of Massachusetts Medical School (UMass) and LogicBio.

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Deanne Eagle

Planet Communications