Myofibrillar myopathy is a rare disease that causes muscle atrophy, stiffness, respiratory muscle weakness, and symptoms such as cardiomyopathy and movement disorders. Patients usually manifest symptoms of the disease during adolescence, but there is currently no effective treatment, and medical costs are extremely high.
A new drug developed by HKBU’s Centre for Chinese Herbal Medicine Drug Development has been designated as an “orphan drug” by the U.S. Food and Drug Administration (FDA) for the treatment of this rare disease, making it the first botanical drug in Hong Kong to receive such designation.
HKBU’s Centre for Chinese Herbal Medicine Drug Development has developed a new drug by extracting the effective components from the traditional Chinese medicinal herb Chaenomeles fructus. HKBU stated that obtaining “orphan drug” status is expected to speed up the review and approval of this new drug, including shortening the review process, waiving new drug launch review fees, and obtaining exclusive market share for seven years after launch. The research team plans to submit a new drug clinical research application to the FDA within two years.
Chaenomeles fructus is the dried mature fruit of Chaenomeles speciosa of the Rosaceae family. It is a widely used clinical Chinese medicine and is often used to treat joint and muscle disorders, such as summer damp-heat paralysis and soreness and pain in waist and knee joints. Although Chaenomeles speciosa bears a similar layperson name in Chinese, it is different from the papaya, which is commonly eaten as a fruit.
Based on clinical treatment cases, the center targets “myofibrillar myopathy” caused by BAG3 gene mutations, combines traditional Chinese medicine theory with modern science and technology, and uses a variety of effective components of traditional Chinese medicinal herb Chaenomeles speciosa to develop a new drug called “CDDs- 2107.”
Expect to Apply to FDA for Clinical Research Within 2 Years
HKBU said that the R&D and production of “orphan drugs” face challenges such as lack of research data, high R&D costs, and small market size. Some countries or regions have, therefore, formulated 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 FDA, pharmaceutical institutions can receive some special incentives and support, such as research funds, extended patent protection period, market exclusivity, and tax incentives.
Professor Bian Zhaoxiang, Associate Vice-President (Development of Traditional Chinese Medicine) and Director of the Centre for Chinese Herbal Medicine Drug Development at HKBU said that it is expected to submit a new drug clinical research application to the FDA within two years. The goal is for CDD-2107 to be approved for marketing by the FDA, thereby Improving the condition of more patients.
