The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending the approval of ruxolitinib cream (Opzelura) for the treatment of non-segmental vitiligo with facial involvement in adults and adolescents from 12 years of age.

“The positive CHMP opinion brings us one step closer to bringing ruxolitinib cream, the first ever treatment for repigmentation in non-segmental vitiligo, to patients and healthcare professionals in the European Union (EU),” Steven Stein, MD, Chief Medical Officer, Chief Medical Officer, Incyte, said in a company news release. “With no centrally approved treatment option currently available in the EU, this positive opinion marks a significant milestone for the vitiligo community.”

The CHMP opinion was based on data from two pivotal Phase 3 clinical trials (TRuE-V1 and TRuE-V2) evaluating the safety and efficacy of ruxolitinib cream versus vehicle (non-medicated cream) in more than 600 people with non-segmental vitiligo, age 12 and older.[1]

Treatment with ruxolitinib cream resulted in significant improvements in facial and total body repigmentation versus vehicle as shown by the number of patients reaching the facial and total body Vitiligo Area Scoring Index (F-VASI-T-VASI) endpoints at Week 24 compared to vehicle, with a higher proportion of patients responding at Week 52.1 The most common adverse reactions (incidence ≥ 1%) were application site acne, application site pruritus, nasopharyngitis, headache, urinary tract infection, application site erythema, and pyrexia.[2]

Results from the TRuE-V program were recently published in The New England Journal of Medicine.

The CHMP’s opinion is now being reviewed by the European Commission, which has the authority to grant centralized marketing authorizations for medicinal products in the EU. If approved, it would be the first approved vitiligo therapy available in the EU indicated for the treatment of non-segmental vitiligo with facial involvement in adults and adolescents from 12 years of age.

References

1 Rosmarin D, et al. Two Phase 3 Randomized Controlled Trials of Ruxolitinib Cream for Vitiligo. New England Journal of Medicine. 2022; 387:1445-1455.
2 Opzelura Prescribing Information. Wilmington, DE. Incyte Corporation.