Small molecule approvals are on the decline in China. According to GlobalData’s Drugs Intelligence Database, from 2020 to 2022, the number of innovator small molecule approvals in China shrank by 21%. However, over the same period, approvals for innovator small molecules treating rare diseases rose by 22%, giving hope to rare disease patients.

According to the GlobalData’s Drug Database, the number of innovator small molecule approvals for rare diseases rose steadily over three years, from three approved in 2019 to 12 last year.

Figure 1 shows that between 2019 and 2021, innovator small molecule approvals for rare diseases rose significantly from just three to 12, displaying a growth of 300%. This trend of increasing approvals looks set to continue into this year, with 11 drugs already approved and 13 in pre-registration with two months left in the year. This trend is likely to continue into the near future, given China’s mature rare disease pipeline.

There are currently 378 drugs indicated for rare diseases under pre-clinical and clinical development, 66% of which are in late-stage development (Phase II to Pre-registration stage). The rise in innovator small molecule approvals for rare diseases since 2019 may be attributed to new regulations set by the Chinese regulatory authority, the National Medical Products Administration (NMPA).

In 2015, China reformed its regulatory system, which introduced review designations, including fast-track programmes to increase drug development for unmet medical needs such as rare diseases and serious conditions. This included the implementation of priority review, breakthrough therapy and accelerated approval designations. The intention of these designations is to drive earlier approval times and faster review times by the NMPA. Since then, 65 innovator small molecules for rare diseases were awarded one of the three designations. Furthermore, in 2020, China established the National Rare Diseases Registry System (NRDRS), which aims to unify rare disease registries into one system and set new protocols for defining rare diseases.

The recent rise in small molecule approvals for rare diseases may be due in part to strong regulatory action by the NMPA combined with a strong rare disease pipeline heavily focused towards late-stage development. In the future, China’s small molecule approvals for rare diseases are likely to continue its rapid growth.