11 January 2024
A research team led by Professor Bian Zhaoxiang, Associate Vice-President (Chinese Medicine Development) and Director of the Centre for Chinese Herbal Medicine Drug Development (CDD) at HKBU, has developed a new drug named "CDD-2107" for the treatment of the rare disease, myofibrillar myopathy. This drug, derived from the Chinese herbal medicine, Chaenomelis Fructus, has been granted orphan drug (a drug used for treating rare disease) designation by the US Food and Drug Administration (FDA), making it the first botanical drug in Hong Kong to receive this qualification.
Myofibrillar myopathy, primarily caused by genetic mutations, is a rare hereditary neuromuscular disorder. Common clinical symptoms include progressive muscle weakness, muscle atrophy, motor impairment, muscle stiffness, respiratory muscle weakness, and cardiomyopathy. Given that there is no effective treatment for this rare disease at present, researchers are still exploring treatment options and evaluating their safety and efficacy.
Based on clinical cases, CDD focused on myofibrillar myopathy caused by BAG3 gene mutation. By integrating traditional Chinese medicine theory with modern scientific technology, and utilising effective components of the commonly used Chinese herbal medicine Chaenomelis Fructus, CDD developed a new drug named "CDD-2107." By applying modern Chinese medicine extraction and purification techniques, the research team identified the most important and effective components from patient's Chinese herbal prescriptions, revealing the potential treatment mechanism of CDD-2107. Results show that CDD-2107 could significantly improve clinical symptoms in patients with myofibrillar myopathy by enhancing muscle strength, improving mobility, and increasing independence, offering new hope to the patients.
Professor Bian said: "CDD-2107 is the fruit of the integration of traditional Chinese medicine theory and modern technology. We plan to submit an Investigational New Drug application to FDA in two years, and hope that CDD-2107 will obtain FDA approval eventually, benefiting more patients."
To learn more about the new drug, please click here for the full press release.
Professor Bian Zhaoxiang (2nd right), Dr. Lin Chengyuan (2nd left), Dr. Hou Mengyang (1st right), and Mr. Duan Zhigang (1st left)
Chaenomelis Fructus