China Medical System Holdings Limited (CMS) has announced a significant step forward in the treatment of vitiligo in China. On October 18, the first prescriptions for ruxolitinib phosphate cream were issued to qualified vitiligo patients in the Greater Bay Area. This follows the approval of the new drug application (NDA) by the Pharmaceutical Administration Bureau (ISAF) of Macau on April 11, 2024, and subsequent approval by the Guangdong Provincial Medical Products Administration on August 19 through the "Hong Kong and Macau Medicine and Equipment Connect" policy. This policy officially introduces ruxolitinib cream for treating non-segmental vitiligo with facial involvement in adults and adolescents aged 12 years and older, offering a novel treatment option within designated medical institutions in the Mainland of the Greater Bay Area.
NDA Acceptance and Real-World Data
In addition to the regional approvals, on September 24, the National Medical Products Administration of China (NMPA) accepted the NDA for the vitiligo indication of ruxolitinib cream. CMS has also conducted a real-world study on ruxolitinib cream in China, in accordance with the regulations of the drug real-world data application pilot program in the Hainan Boao Lecheng International Medical Tourism Pilot Zone. The study results demonstrated positive efficacy, consistent with key outcomes from global pivotal clinical studies. All secondary efficacy endpoints showed a trend of benefit consistent with the primary efficacy endpoint, with the treatment effect for vitiligo continuing to improve with longer treatment duration. Safety monitoring data from the Pilot Zone identified no new safety events, with adverse events mostly being grade 1 or 2 in severity. No adverse event led to discontinuation or withdrawal, and no serious adverse event related to the study drug occurred.
Potential Impact on Vitiligo Treatment in China
If ruxolitinib cream is successfully approved for marketing in Mainland China, it will be the first prescription drug approved by the NMPA for repigmentation in vitiligo. This would provide new hope for Chinese vitiligo patients, addressing a significant unmet medical need. It is estimated that there are approximately 14 million vitiligo patients in China, with non-segmental vitiligo accounting for about 85% of cases. Current treatments, such as topical corticosteroids and calcineurin inhibitors, have clinical deficiencies, including long-term adverse reactions and limited efficacy.
Ruxolitinib Cream: Mechanism and Approvals
Ruxolitinib cream (Opzelura) is a novel cream formulation of Incyte’s selective JAK1/JAK2 inhibitor, ruxolitinib. It is approved by the U.S. Food & Drug Administration (FDA) for the topical treatment of nonsegmental vitiligo in patients 12 years of age and older, and is the first and only treatment for repigmentation approved for use in the United States. The cream is also approved in the U.S. for the topical short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis (AD) in non-immunocompromised patients 12 years of age and older whose disease is not adequately controlled with topical prescription therapies, or when those therapies are not advisable. In Europe, ruxolitinib cream is approved for the treatment of non-segmental vitiligo with facial involvement in adults and adolescents from 12 years of age.
CMS and Future Directions
CMS has secured an exclusive license to develop, register, and commercialize ruxolitinib cream in Mainland China, Hong Kong Special Administrative Region, Macau Special Administrative Region, Taiwan Region, and eleven Southeast Asian countries through a collaboration and license agreement with Incyte. CMS is also actively working on transferring ruxolitinib cream production to domestic facilities to expedite its availability in China. The company remains committed to meeting unmet medical needs by bringing innovative pharmaceutical products to Chinese patients.
