A rare offer

The definition of a rare disease varies from country to country. In the US it is defined as a condition that affects fewer than 200,000 Americans, while in the European Union the threshold is set at an incidence of five in 10,000. The latest unofficial metric in China is one in 10,000 for newborns or an overall caseload of less than 140,000.

This means that Europe and the US recognise some 7,000 diseases as rare. China, which is only beginning to address the treatment of rare diseases, uses an official list which categorises 121 illnesses for their rarity.

Yet China, by dint of its huge population, is believed to have the largest numbers of rare disease patients in the world – making it a huge market for companies able to address their needs. One such firm is CANbridge Pharmaceuticals (or Beihai Kangcheng) which listed in Hong Kong last month.

CANbridge was founded by an alum from Peking and Brown universities called James Qun Xue in 2012 to focus on the treatment of rare diseases such as glioblastoma (an aggressive form of brain cancer), hunter syndrome (a genetic disease that inhibits the breakdown of sugars) and haemophilia A.

CANbridge is part of a new wave of Chinese ‘biologics’ firms – i.e developing pharmaceuticals derived from biological as opposed to chemical sources. Innovent, Beigene, Wuxi Biologics and Zai Lab – all founded about 10 years ago by returnees from the US to China – are all part of the same wave.

CANbridge noted in its prospectus that it has developed a comprehensive pipeline of 13 drug assets with “significant market potential”, including three marketed products.

Until recently rare diseases in China were largely ignored by the state health system and pharmaceutical companies, which focused more on treating illnesses with bigger caseloads, where the largest impact could be achieved with treatments.

A few years ago, however, there was a shift as public anger began to mount over the shortages of drugs for rare cancers and genetic conditions – especially as it was possible to buy cheaper generic versions of many similar drugs in India (see WiC417). This prompted Beijing to overhaul its policy forcing Chinese companies to slash their drug prices in exchange for large state procurement contracts. The government also encouraged drugmakers to innovate – to invent new drugs that might be exported around the world instead of manufacturing generics for local use or producing pharmaceutical ingredients for foreign drugmakers.

In November 2019 that policy began to pay off when the US approved its first Chinese-developed cancer drug – Brukinsa, a treatment for mantle cell lymphoma, created by Beigene.

A second driver in China’s push for better rare disease treatment is the falling birth rate. Most rare diseases are genetic and first appear in childhood. For parents, caring for a sick child can mean years of trying to get a diagnosis and the risk of bankruptcy in funding treatment.

Beijing hopes to encourage more families to have more children – one of the reasons for offering better care for rare childhood illnesses. And at the other end of the spectrum China has a rapidly aging population that will demand better healthcare than its predecessors.

And then there is the Healthy China 2030 campaign that seeks to improve the general health of the population by the end of the decade with the help of new and locally made treatments.

The rare disease drug market in China is expected to grow from $1.3 billion in 2020 to $25.9 billion in 2030, Frost and Sullivan has estimated.

According to the Economist Intelligence Unit, China’s list of 121 rare diseases applies to three million people. CANbridge estimates there are 120,000 Chinese suffering from Haemophilia A, for instance.

CANbridge listed its shares at a price of HK$12.18, raising HK$604 million ($77.5 million). The IPO included General Atlantic and Wuxi Biologics among its cornerstone investors. However, the share have struggled since the listing, trading this week below the HK$8 level. Perhaps its rare disease therapies are still to capture the interest of the wider market.

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