OBJECTIVES

: Gene therapies (GTs) are transformative therapies with potential to cure serious and chronic diseases. Europe plays a critical role in the research and development of GTs, and implemented regulation pathways to promote the market authorization of innovative therapies. Asian countries, especially Japan, South Korea and China, are processing rapidly to increase their global competencies in this field. This study aims to compare the currently approved GTs in Europe, Japan, South Korea and China.

METHODS

: The official websites of European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA) in Japan, Ministry of Food and Drug Safety (MFDS) in South Korea, and National Medical Products Administration (NMPA) in China, were searched to identify the gene therapies granted marketing authorization until February 2020.

RESULTS

: A total of 7 GTs were approved in Europe, among which Glybera® was withdrawn since October 2017. Both Japan and China had two approved and marketed GTs. The first and only GT approved in South Korea, Invossa®, was suspended from sale due to the false reporting of product component. Kymriah® was approved in Europe and Japan in August 2018 and March 2019, respectively. However, Novartis stated that the commercialization of Kymriah® in Japan would be ready in the second half of 2020 because they struggled to improve the manufacturing capacity of domestic Japanese supplier. Kymriah® holds great possibility to be approved as the first GT in South Korea and China. In China, Novartis entered into collaboration agreement with Chinese manufacturer Cellular Biomedicine (CBMG) to manufacture and supply Kymriah® in September 2018. Kymriah® was designated as orphan drug by South Korea in March 2019 to accelerate its approval.

CONCLUSIONS

: Europe is ahead of Asian countries with more GTs approved, while Asian countries are exploring proactive strategies to foster innovative GTs market access.