The Hong Kong Baptist University (HKBU) Chinese Medicine Innovation and Development Centre has developed a new drug to treat a rare disease called "myofibrillar myopathy" by extracting an effective component from the Chinese medicine "papaya crinosa". The drug has been granted "orphan drug" status by the US Food and Drug Administration, becoming the first botanical drug in Hong Kong to receive this status.

The designation of "orphan drugs" (drugs used to treat rare diseases) is expected to speed up the approval of new drugs, including shortening the evaluation process, waiving the evaluation fees for new drugs, and obtaining exclusive market rights for seven years after marketing. The research team plans to submit an investigational application for a new drug to the U.S. Food and Drug Administration within two years.

There is no effective treatment for myofibrillar myopathy

Myofibromyopathy is caused by genetic mutations, including mutations in the BAG3 gene, and is a rare hereditary neuromuscular disease. Its symptoms are similar to muscular dystrophy, usually in the adolescent years, the common clinical symptoms are very serious, including progressive muscle weakness, muscular dystrophy, movement disorders, muscle stiffness, respiratory muscle weakness and cardiomyopathy.

There is currently no effective treatment for the disease, and the medical costs are very high, placing a heavy burden on patients, families and society. It is a rare disease, and researchers are still exploring treatment options for it and evaluating its safety and effectiveness.

Based on clinical treatment cases, the Chinese Medicine Innovation Research and Development Center has combined traditional Chinese medicine theory with modern science and technology for myofibromyopathy caused by BAG3 gene mutation, and adopted various active ingredients of Chinese herbal medicine "wrinkled papaya" to develop a new drug named "CDD-2107".

A new Chinese medicine was developed from the effective components of Papaya ruticola, which is the dried and ripe fruit of the rosaceae, and is commonly used in the treatment of joint muscle diseases, such as damp-heat arthralgia and pain of waist and knee. Papaya with wrinkled skin and generally as a fruit to eat papaya different, the latter called papaya, papaya family papaya genus.

The research team based on the theory of traditional Chinese medicine, from the clinical effect, the use of modern Chinese medicine extraction and purification technology for research. They identified the most important active components from patients' TCM prescriptions and revealed the possible mechanism of action of CDD-2107. The results of the study show that CDD-2107 can significantly improve the clinical symptoms of myofibrillar myopathy patients, including increased muscle strength, as well as improved mobility and independence, providing new hope for patients.

Professor Ian Siu-cheung, Associate Vice President (Chinese Medicine Development) and President of HKBU's Chinese Medicine Innovation Research and Development Centre, said: "CDD-2107" is the crystallization of the integration of traditional Chinese medicine theory and modern technology. Its orphan drug designation by the US Food and Drug Administration is an encouraging milestone, reflecting the recognition of the team's dedication and scientific innovation.

In the face of challenges such as lack of research data, high research and development costs and small market size, some countries or regions have adopted policies and measures to encourage pharmaceutical companies to develop and produce drugs for the treatment of rare diseases. After a drug is approved as an orphan drug by the U.S. Food and Drug Administration, pharmaceutical organizations can receive special incentives and support, such as research funding, extended patent protection, market exclusivity, and tax incentives.

Professor Bian said: "We expect to submit a new investigational drug application to the US Food and Drug Administration within two years, which is an important step towards CDD-2107 becoming an internationally recognized safe and effective drug for the treatment of myofibrillar myopathy caused by BAG3 gene mutation." Our ultimate goal is for CDD-2107 to be approved by the U.S. Food and Drug Administration to improve outcomes for more patients."