The Chinese biopharmaceutical industry: the renaissance of small molecules in rare diseases

Small molecule approvals are down in China. According to GlobalData’s Drugs Intelligence database, from 2020 to 2022, the number of innovative small molecule approvals in China decreased by 21%. However, over the same period, approvals of innovative small molecules treating rare diseases increased by 22%, giving hope to rare disease patients.

According to GlobalData’s drug database, the number of approvals of innovative small molecules for rare diseases has increased steadily over three years, from three approved in 2019 to 12 last year.

Figure 1 shows that between 2019 and 2021, approvals of innovative small molecules for rare diseases increased significantly, from just 3 to 12, showing a growth of 300%. This trend of increasing approvals is expected to continue this year, with 11 drugs already approved and 13 in pre-registration with two months remaining in the year. This trend is expected to continue in the near future, given China’s mature rare disease pipeline.

There are currently 378 drugs indicated for rare diseases in preclinical and clinical development, of which 66% are in advanced development (phase II to pre-registration phase). The increase in approvals of innovative small molecules for rare diseases since 2019 can be attributed to new regulations established by China’s regulatory authority, the National Medical Products Administration (NMPA).

In 2015, China reformed its regulatory system, which introduced review designations, including accelerated programs to increase drug development for unmet medical needs such as rare diseases and serious conditions. This included the implementation of Priority Review, Breakthrough Therapies, and Fast Track Approval Designations. The intent of these designations is to expedite approval times and NMPA review times. Since then, 65 innovative small molecules for rare diseases have received one of three designations. In addition, in 2020, China established the National Rare Disease Registry System (NRDRS), which aims to unify rare disease registries into a single system and establish new protocols for defining rare diseases.

The recent increase 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 on late-stage development. Going forward, small molecule approvals in China for rare diseases are expected to continue their rapid growth.

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