The current treatment landscape for Fabry disease in China is completely dependent on enzyme replacement therapies (ERTs) developed by western companies like Sanofi (Fabrazyme) and Takeda (Replagal). Due to the limited affordability of these therapies, there exists a substantial unmet clinical need that could be addressed by newer therapies. However, only a few western innovator companies conducted clinical trials for new Fabry disease pipeline therapies in China, despite the country having the highest number of diagnosed prevalent cases of Fabry disease among the 16 major markets (16MM*), says GlobalData, a leading data and analytics company.

According to GlobalData’s Pharmaceutical Intelligence Center, China holds the highest number of diagnosed prevalent cases of Fabry disease among the 16 major markets (16MM*). The diagnosed prevalent cases of Fabry disease in the country are expected to increase from 10,151 in 2023 to 10,462 in 2028, recording an annual growth rate (AGR) of approximately 3%.

Prashant Khadayate, Pharma Analyst at GlobalData, comments: “Due to the limitations of ERTs, such as the high cost of treatment (Fabrazyme and Replagal are listed among the top five most expensive originator brands in China), potential immune reactions, and need for intravenous infusion, there remains a substantial unmet clinical need for oral drugs in Fabry disease treatment in China.”

AceLink Therapeutics, a US-based clinical-stage drug development company developing an oral substrate reduction therapy (SRT) called AL-1211, initiated a Phase II trial in China for Fabry disease in August 2023. In the past, only Sanofi and Takeda conducted clinical trials in China. Moreover, Australia and Japan are more popular locations to conduct clinical trials for Fabry disease within the Asia-Pacific region.

Khadayate concludes: “Innovator foreign companies preferred other locations over China, irrespective of the substantial Fabry disease patient population. This indicates that there may be challenges in the execution of clinical trials in Fabry disease in this country. As the rare disease policy landscape in China is evolving rapidly, further collaboration between the Chinese government and western pharmaceutical companies would encourage the latter to make investments in the execution of clinical trials. This would also help ensure that Chinese patients get quick access to innovative therapies and would not need bridging trials to get approval in China.”

*16MM: Australia, Brazil, Canada, China, France, Germany, Italy, Japan, Mexico, Spain, South Africa, South Korea, the UK, and the US.