Back to Press ReleaseCANbridge Announces the First Prescription of Gaurunning®, a Class 1 New Drug for Treating Type I and III Gaucher Disease
Suzhou, China, July 2, 2025 – CANbridge Pharmaceuticals Inc. (1228.HK), a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies to treat rare diseases, announced today that Gaurunning® (velaglucerase-beta for injection), a long-term enzyme replacement therapy for adolescents (12 years and older) and adults with type I and III Gaucher disease, which was approved for marketing on May 13, has been prescribed for the first time at Xinhua Hospital Affiliated to Shanghai Jiaotong University School of Medicine. This marks the official entry of the first domestically independently developed enzyme replacement therapy into clinical use in China.
A 14-year-old patient from Xinjiang became the first to receive Gaurunning treatment. Diagnosed with type I Gaucher disease at the age of 9, the patient is currently experiencing severe symptoms and in urgent need of treatment. The clinical application of Gaurunning opens a new therapeutic option for domestic Gaucher disease patients.
According to Zhang Huiwen, Chief Physician of the Department of Pediatric Endocrinology and Genetics at Xinhua Hospital Affiliated to Shanghai Jiaotong University School of Medicine, Gaucher disease is a rare autosomal recessive metabolic disorder caused by a functional defect of glucocerebrosidase in lysosomes. Clinical manifestations include hepatosplenomegaly, anemia, bone pain, and neurological lesions, which may even endanger life due to complications in severe cases. Velaglucerase-beta for injection (Gaurunning) can specifically supplement the deficient glucocerebrosidase in the lysosomes of Gaucher disease patients through intravenous infusion. Among the approved Gaucher disease drugs in China, Gaurunning has the most comprehensive indication coverage, applicable to both type I and III Gaucher disease patients, as well as to subpopulations of adolescents and adults who are unresponsive or intolerant to other drug treatments.
Gaucher disease is classified into three subtypes based on neurological involvement and progression rate: non-neuronopathic (type I), acute neuronopathic (type II), and chronic or subacute neuronopathic (type III). The majority of Gaucher disease patients are type I and III. Pediatric patients often have more severe symptoms, and early diagnosis with active enzyme replacement therapy can significantly improve the prognosis. Clinical data show that Gaurunning significantly improves core indicators of Gaucher disease. After 9 months of treatment, subjects experienced a 35.32% reduction in spleen volume and a 2.17 g/dL increase in hemoglobin.
The issuance of the first prescription for Gaurunning has brought new hope to the Gaucher disease patient community. It also demonstrates the outstanding strength of the Department of Pediatric Endocrinology, Genetics and Metabolism of Xinhua Hospital in promoting medical technological innovation and the translation of research achievements.
About CANbridge Pharmaceuticals Inc.
CANbridge Pharmaceuticals Inc. (HKEX:1228) is a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare disease. CANbridge has a differentiated drug portfolio, with 3 approved drugs and a pipeline of 7 assets, targeting prevalent rare disease indications that have unmet needs and significant market potential. These include Hunter syndrome and other lysosomal storage disorders, complement-mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases. The CANbridge Next-Generation Innovation and Process Development Facility is developing novel, potentially curative, gene therapies for rare genetic diseases, including Pompe disease, Fabry disease, spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD) and other neuromuscular conditions, and collaborates with world-leading researchers and biotech companies. CANbridge global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, UMass Chan Medical School, the University of Washington School of Medicine and Scriptr Global.
For more on CANbridge Pharmaceuticals Inc., please go to: www.canbridgepharma.com.
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