Small molecule approvals for rare diseases in China recorded a staggering 300% growth from just 3 to 12 between 2019 and 2022 due to swift regulations set by the National Medical Products Administration (NMPA). As a result, the trend of increasing approvals had now reached all time-highs in 2022, says GlobalData, a leading data and analytics company.

GlobalData’s Drugs Database reveals that there are 12 drugs approved in 2022 with 13 drugs in pre-registration with a month left in the year.

Currently, there are 378 small molecules treating rare diseases under clinical and preclinical development in China. Approximately 66% of these drugs are in late-stage development (Phase II to Pre-registration), suggesting that a large cohort of drugs is set to be approved in the near-term.

Kevin Marcaida, Pharma Analyst at GlobalData, comments: “In 2015, China reformed its regulatory system to introduce review designations including fast-track programs 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 small molecules treating rare diseases were successfully awarded a regulatory designation. 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.

Marcaida concludes: “The rise in small molecule approvals for rare diseases in China will likely reach greater heights. China boasts a growing rare disease pipeline heavily focused towards late-stage development, alongside the NPMA proactively improving regulatory standards to speed up approval times.”