Prior to licensing trofinetide ROW rights to Acadia, Neuren...

Prior to licensing  trofinetide ROW rights to Acadia, Neuren highlighted the potential and identified populations of Rett individuals across various ex-US jurisdictions.

The largest potential population (28,000) was in urban China.



As explained in this post in early 2021, China established its first Rare Disease List in 2018. One of the criteria for inclusion in this list was that the rare disease had to be treatable and intervention had to be feasible and economically accessible. Alternatively, if there was no currently available treatment, a disease was eligible if it was being investigated under a government-funded research program.

There were 121 rare diseases included in the 2018 list but Rett syndrome was not one of them.

Last month, the Chinese Government National Health Commission announced its second Rare Disease List, which includes a further 86 rare diseases. This time, both Rett syndrome and Fragile-X syndrome have been included.

Numerous regulatory incentives exist for rare disease drugs to be launched in China. Those eligible for priority review and approval receive regulatory review within 130 days which is further shortened to just 70 days for urgently needed rare disease drugs that have been previously approved abroad. In addition, it is possible to gain approval with overseas clinical data, regardless of whether the drug has been approved in other countries or not.  Finally, like in the United States, there is an orphan drug marketing exclusivity period of 7 years.

Getting a rare disease on the national list in China is considered to be a starting point for gaining momentum in creating better diagnostics, expanding insurance coverage, generating public awareness and strengthening doctors' understanding of the disease.

The opportunity is now there; I guess it really comes down to whether Acadia is interested.


https://www.canbridgepharma.com/en/media-news/press-release/20230921/
https://www.chinadaily.com.cn/a/202309/26/WS651233bda310d2dce4bb7d05.html
https://www.frontiersin.org/articles/10.3389/fphar.2023.1223056/full