Following 2 phase 3 trials, the European Medicines Agency Committee for Medicinal Products for Human Use gives positive opinion on the use of ruxolitinib cream as a treatment for facial-involved non-segmental vitiligo.
Global pharmaceutical company Incyte announced an optimistic outlook on ruxolitinib cream (Opzelura) as an effective treatment for adolescent and adult patients with vitiligo.
The opinion was issued by the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CMPH) following the completion of two controlled Phase 3 trials1. The trials (TRuE-V1 and TRuE-V2), which involved 674 participants as young as age 12, were initially published in the New England Journal of Medicine in October 2022.
Researchers conducted the double-blind trials in North America and Europe. Participants were patients with non-segmental vitiligo and depigmentation in at least 10% of their body’s surface area. The trials looked primarily at vitiligo with facial involvement.
Over a 24-week span, a ratio of 2:1 patients applied either a 1.5% ruxolitinib cream or the control treatment at a twice-daily rate. For the remainder of the trial (28 weeks), all patients, including those who had previously been assigned to the control treatment, were asked to use the ruxolitinib cream.
Throughout the duration of the trials, researchers sought out endpoints which included an overall decrease of 75% in accordance with the Facial Vitiligo Scoring Index (F-VSI/F-VSI75) and improvement as measured by the Vitiligo Noticeability Scale (VNS).
Nearly 30% of all TRuE-V1 participants (n=330) had demonstrated F-VSI75 improvement by week 24. In TRuE-V2, 31% of total participants (n=344) saw similar improvement by the same time mark.
More than 50% of participants in each respective trial experienced adverse events, including acne, itching and nasal inflammation. Despite this, researchers said ruxolitinib cream demonstrated superiority to the control cream.
“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,” said Incyte Chief Medical Officer Steven Stein, MD, in a February press release2. “With no centrally approved treatment option currently available in the EU, this positive opinion marks a significant milestone for the vitiligo community.”
The U.S. Food and Drug Administration (FDA) approved the use of 1.5% ruxolitinib cream in July 2022, when it became the first, and only, FDA-approved repigmentation treatment for non-segmental vitiligo. Currently, ruxolitinib cream is also FDA-approved to treat other dermatological conditions such as atopic dermatitis.
With a positive opinion of the cream, patients living with vitiligo and melanoma-associated vitiligo (which affects 3% of people with melanoma)3 in European Union countries may soon have access to at-home treatment for the very first time.