Beijing, China; Cambridge, Mass., September 21, 2023 — CANbridge Pharmaceuticals Inc. (HKEX:1228), a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative rare disease and rare oncology therapies, is pleased to share news of the publication of China’s “Second List of Rare Diseases.” This significant development stands a pivotal moment in China’s healthcare landscape and holds substantial importance for the sector at large.

In significant development, the National Health Commission, jointly with the Ministry of Science and Technology, the Ministry of Industry and Information Technology, the National Medical Products Administration, the National Administration of Traditional Chinese Medicine and the Logistic Support Department of the Central Military Commission, has unveiled the “Second List of Rare Diseases” on September 18, 2023, after the National Health Commission had launched the selection of rare diseases to be included in the second list in February 2022. This follows the release of China's First List of Rare Diseases in May 2018, signifying a pioneering step in addressing rare diseases within the nation.

The First List of Rare Diseases, comprising 121 rare diseases, was a historic milestone as it marked the initial attempt by the Chinese government to define and catalog rare diseases. The list served as a crucial reference point for formulating policies related to rare diseases, impacting approximately 3 million patients across China, an estimation based on the published epidemiological data.

The newly released Second List of Rare Diseases now features 86 rare diseases spanning 17 disciplines, including Hematology, Dermatology, Rheumatology and Immunology, Pediatrics, Neurology, and Endocrinology among others. Notable inclusions are Alagille syndrome (ALGS), congenital biliary atresia (BA), and glioblastoma (GBM). With this addition, China’s rare disease catalog now comprises a total of 207 rare diseases across both lists.

Rare diseases, characterized by their extraordinarily low incidence and prevalence rates, collectively affect an estimated 250 million to 300 million patients worldwide, with approximately 30 million affected individuals in China. At present, there are 7,000 known rare diseases in the world. Alarmingly, 95% of these rare diseases lack effective treatment options, with 80% classified as congenital genetic diseases. Half of rare disease patients are children, and 30% tragically succumb to these conditions before reaching the age of 5.

Globally, the definition of rare diseases varies. The World Health Organization classifies rare diseases as diseases as those affecting 0.65‰ to 1‰ of the total population, while the United States considers diseases rare if they affect fewer than 200,000 patients in nationwide or if the expected therapeutic drug sales in the country are insufficient to cover R&D costs. The European Union, meanwhile defines diseases as rare if they have an incidence rate of less than 0.5‰ in the region. China, on the other hand, adopts a list-based approach to identify rare diseases, underscoring the momentous importance of the Second List of Rare Diseases.

Second List of Rare Diseases

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 and rare oncology. CANbridge has a differentiated drug portfolio, with 4 approved drugs and a pipeline of 10 assets, targeting prevalent rare disease and rare oncology 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, as well as glioblastoma multiforme. 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) and other neuromuscular conditions, and collaborates with world-leading researchers and biotech companies. Animal data from the SMA gene therapy was presented in 2022 at the American Society for Gene and Cell Therapy (ASGCT), the European Society for Gene and Cell Therapy (ESGCT) and the World Muscle Congress. 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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CANbridge Pharmaceuticals Inc.

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