According to a press release from Incyte on April 20, the European Commission (EC) announced that it has granted marketing authorization to Opzelura 1.5% (ruxolitinib) cream for the treatment of non-segmental vitiligo with facial involvement in individuals aged 12 years and older. Non-segmental vitiligo is the most common form of vitiligo, characterized by depigmented patches on both sides of the body surface.
Ruxolitinib is a selective janus kinase (JAK) inhibitor targeting both JAK1 and JAK2 subtypes, acting by inhibiting intracellular signaling of various immune-related cytokines and growth factors. Ruxolitinib is administered topically as a 15 mg/g (1.5%) cream for the treatment of non-segmental vitiligo and is the first and only topical JAK inhibitor approved for use in the EU.
In patients with non-segmental vitiligo affecting the face, Opzelura is approved for topical application twice daily to areas of depigmented skin, up to 10% of the body surface area. For satisfactory hyperpigmentation, consistent use of Opzelura for at least 24 weeks is required.
Opzelura is approved in the United States for the topical treatment of non-segmental vitiligo in patients aged 12 years and older, and for the short-term, non-continuous chronic topical treatment of mild to moderate atopic dermatitis (AD) in patients aged 12 years and older whose disease is not adequately controlled by topical prescription therapies, or when such therapies are not advisable.
Currently, ruxolitinib is available in oral tablet form (Jakavi) for the treatment of myelofibrosis, polycythemia vera, and graft-versus-host disease.
NICE guidelines on topical ruxolitinib for the treatment of non-segmental vitiligo are under development and are expected to be published in February 2024.
EC's decision was based on data from two pivotal phase 3 clinical trials (TRuE-V1 [NCT04052425] and TRuE-V2 [NCT04057573]), evaluating the efficacy and safety of Opzelura versus excipients (non-pharmacological creams) in over 600 patients aged 12 years and older with non-segmental vitiligo.
Results from the TRuE-V study showed that patients treated with Opzelura experienced significant improvements in facial and systemic repigmentation compared to controls, as indicated by the number of patients reaching the facial and systemic vitiligo area score index (F-VASI-T-VASI) endpoints at week 24 compared to the excipient, and open-label expansion at week 52.
The results at week 24 were consistent across both studies, showing that 29.8% and 30.9% of patients receiving Opzelura achieved a ≥75% improvement from baseline in the primary endpoint, Facial Vitiligo Area Score (F-VASI75), compared to 7.4% and 11.4% of patients receiving excipient-based treatments in TruE-V1 and TruE-V2, respectively. By week 52, approximately half of the patients treated with Opzelura had achieved F-VASI75.
Furthermore, at week 24, over 15% of patients receiving Opzelura achieved a ≥90% improvement from baseline in F-VASI (F-VASI90), compared to approximately 2% of patients receiving excipient-based treatments. By week 52, approximately one-third of the patients treated with Opzelura had achieved F-VASI90.
Regarding safety, no serious treatment-related adverse events were associated with ruxolitinib cream; the most common adverse reaction was application-site acne.
