Livmarli mentioned as a case study The Policy Address highlights Hong Kong's strengths in research and development, emphasizing its capcity to independently approve new drugs Rare disease drugs will receive significant benefits  


Hong Kong – 14 November, 2023— 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 disease and rare oncology.


Hong Kong's Chief Executive, John Lee Ka-chiu, delivered his second Policy Address at the end of October, in which he addressed the drug registration system and its impact on healthcare. He highlighted Hong Kong’s strength in scientific research, particularly in the context of independently approving new drugs. As a results, it is expected that medicines for rare diseases will be benefited, the following is a related report by HK01:


The Healthcare Bureau is set to introduce the new "1+" mechanism next month, which will enable the registration of serious or rare diseases drugs in Hong Kong, facilitated through a certificate for a pharmaceutical product, along with an expert report and local data. The Secretary for Health, Professor Lo Chung-mau, stated that he would not assess the number of drugs to be registered in Hong Kong under the "1+" mechanism, but believed the measure would attract more and more pharmaceutical companies to develop drugs in Hong Kong. He emphasized that Hong Kong has the advantage in scientific research to develop its own clinical approvals system without the need to follow mainland China or the United States. He also reiterated that the establishment is not for the sake of doing, but for the health of the public. The "1+" mechanism is the first step towards the establishment of the Hong Kong Centre for Medical Products Regulation(CMPR), which Hong Kong can adopt the "primary evaluation" approach in approving applications for registration of pharmaceutical products in the future without the need for them to be endorsed by other drug accreditation bodies. 


When asked about his confidence in the establishment of the CMPR during the current term, he stated that it would depend on the effectiveness of the "1+" mechanism after its implementation. He emphasized that the government would adopt a pragmatic approach rather than engaging in actions for the sake of it. Additionally, he highlighted that Hong Kong has always been a leader in healthcare rather than a follower. Despite the relatively small number of patients, market size, and clinical volume in Hong Kong, he pointed out that the region possesses its own strengths. He also expressed his belief that with the clinical trials conducted by the Research and Development Institute of the Loop, more pharmaceutical companies would choose to develop drugs in Hong Kong once the "primary evaluation” is in place.


He further pointed out that "Hong Kong's version of Food and Drug Administration" is often used to simulate the CMPR, but he disagreed with this, as Hong Kong has the advantage of conducting scientific research to establish its own clinical approvals system. It does not need to strictly follow the regulations of Mainland China or the United States. Additionally, he mentioned that Hong Kong's regulatory organizations are not responsible for overseeing the food sector. He hoped that after the establishment of the Centre, the Hong Kong Government would become a member of the International Council for Harmonisation (ICH). He anticipated that it would take approximately eight to ten years for Hong Kong to attainthe ICH membership, and he believed that the Hong Kong Government would require a few years of experience in the "primary evaluation" before pursuing membership.


The Director of Health, Ronald Lam Man-kin, added that the "primary evaluation" could not be achieved in one go, as the drugs involved extensive data analysis and approval procedures, including raw data from the clinical development stage. The implementation of the "1+" mechanism at the moment would enable the Government to accumulate certain experience to determine the necessary experts and mechanisms were required for the "primary evaluation", which would be implemented only when the ancillary facilities were perfected.


He also highlighted that besides accumulating experience for the "primary evaluation", the "1+" mechanism can expedite the introduction of new drugs for rare or serious diseases in Hong Kong. Using Alagille Syndrome as an example, he explained that the new drug Maralixibat (Livmarli) currently requires two Certificates of Pharmaceutical Product before it can be registered in Hong Kong, which takes two years to complete. However, with the implementation of the "1+" system, the drug can be approved and introduced in Hong Kong within approximately five months. This significantly reduces the time by about 17 months compared to the original period, will enable timely medical treatment for patients.


Extracted from: HK01 | By: L. Y. Kong

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LIVMARLI® (maralixibat) oral solution is a minimally absorbed ileal bile acid transporter (IBAT) inhibitor that blocks the enterohepatic circulation of bile acids, reduces bile acid levels in the liver and serum, reduces the resultant liver injury and relieves pruritus. LIVMARLI is the first approved drug to treat cholestatic pruritus associated with Alagille syndrome in the US, Canada, Europe and China.  

LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including biliary atresia.  LIVMARLI has been granted Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and biliary atresia by the FDA.


About Alagille Syndrome (ALGS)

Alagille syndrome (ALGS) is an autosomal dominant, multisystem disorder which can lead to end-stage liver disease and death. The birth prevalence of ALGS is between 1/30,000 and 1/50,000 [1], and it has been registered in the National Rare Diseases Registry System of China (NRDRS). ALGS has been included in the “Second National List of Rare Diseases” in China in September 2023.The disease is characterized by a paucity of bile ducts and involvement of extrahepatic organs, such as the kidneys and eyes, as well as bones and the cardiovascular system. All patients experience liver involvement [2, 3], which often manifests as chronic cholestasis (slowed or stalled bile flow), usually in the neonatal period or within the first 3 months after birth. In addition to jaundice, skin xanthoma, and hepatomegaly, patients may also experience severe pruritus [4], which can lead to skin disfigurement, emotional disorders, sleep deprivation, and interruption of school learning, due to scratching in affected children [5]. It seriously affects the growth, development, and quality of life of patients [6] and can lead to liver transplantation [7].

  1. Kamath et al, JPGN 2018; 67: 148-156
  2. Turnpenny PD, Ellard S. Eur J Hum Genet, 2012;20:251–57.
  3. Saleh M, et al. Appl Clin Genet, 2016;9:75–82.
  4. Elisofon SA, et al. J Pediatr Gastroenterol Nutr 2010; 51:759–765.
  5. Elisofon et al. JPGN. 2010;51: 759-765.
  6. Abetz-Webb et al. Hepatology. 2014, 60(4), 526-527.
  7. Kamath BM, et al. Hepatol Comms 2020; 4:387–398.


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 four 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.

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