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Ruxolitinib cream approved by FDA for vitiligo

Ruxolitinib cream has become the first and only FDA-approved product for re-pigmentation in non-segmental vitiligo

Ruxolitinib cream has been approved by the US Food and Drug Administration for the treatment of non-segmental vitiligo according to a press release by the manufacturer Incyte.

Vitiligo is a common, autoimmune skin disorder, which results from the loss of melanocytes in the epidermis and which presents clinically as well-demarcated, white patches on the body. The condition has an estimated prevalence of 0.5 – 2% of the worldwide population. Vitiligo is classified into two main forms, non-segmental and segmental. The non-segmental form (also referred to as bilateral or generalised vitiligo) is symmetrical, whereas segmental vitiligo, is more localised. Non-segmental vitiligo is the more common form and accounts for 85 – 90% of cases. Ruxolitinib is a Janus kinase inhibitor and the cream has already showed anti-inflammatory and prompt antipruritic effects in the management of patients with atopic eczema.

The FDA approval of ruxolitinib (brand name Opzelura™) was based on the results of two clinical trials, TRuE-V1 and TRuE-V2 . The two trials were similar in design and sought to evaluate the efficacy and safety of ruxolitinib cream in adolescent (12 years and older) and adult participants with non-segmental vitiligo for whom the vitiligo involved area (facial and non-facial) did not exceed 10% of their body surface area (BSA). In both trials, participants were randomised received ruxolitinib cream 1.5% or vehicle for 24 weeks and which was applied twice daily. Following on from the initial 24 week period, participants were offered the opportunity to continue in the treatment extension arm for up to 52 weeks. The primary endpoint for both trials was the facial Vitiligo Area Scoring Index (F-VASI75) and a 75% improvement from baseline in the F-VASI (F-VASI75) has been identified as within-patient clinically meaningful thresholds.

Ruxolitinib cream: clinical efficacy

A dose ranging phase 2 randomised trial did provide evidence for the effectiveness of the treatment. In the trial, 157 patients (mean age, 48·3 years, 46% male), with depigmentation of 0·5% or more of their facial body surface area (BSA) and 3% or more of their non-facial BSA, were randomised 1:1:1:1 to receive ruxolitinib cream (1·5% twice daily, 1·5% once daily, 0·5% once daily, or 0·15% once daily) or vehicle twice daily. The primary endpoint was a F-VASI50 after 24 weeks and this was achieved after 24 weeks by 45% of patients using the 1.5% cream twice daily and 50% of those using the same strength once daily compared to only 3% receiving placebo.

Although the results of TRuE-V1 and V2 are yet to be fully published, the manufacturer press release reports that after 24 weeks, approximately 30% of patients using ruxolitinib cream achieved the primary endpoint compared to approximately 8% of those using the vehicle in TRuE-V1 and 13% in TRuE-V2. Additionally, after 24 weeks, more than 15% of those receiving ruxolitinib cream achieved a F-VASI90 (i.e., 90% improvement from baseline), compared to approximately 2% of patients treated with vehicle.  At week 52, approximately 50% of ruxolitinib cream patients achieved the primary endpoint and 30% had achieved a F-VASI90.

Although ruxolitinib cream has yet to be approved by the European Medicines Agency, it is currently under review by the organisation.

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