Vitiligo
Overview
- Vitiligo is a common condition where an overactive immune system destroys the skin pigmentation. Globally, it affects around 1% of people.
- The disease has a large societal and economic burden.1,2
- At present, there are no Food and Drug Administration-approved treatments that can deliver re-pigmentation, and treatment relies on topical steroids and calcineurin inhibitors, UV therapies, bleaching agents, or simply make-up and camouflage.
Immune pathogenesis
- Vitiligo is driven predominately by overactivation of the Th1 pathway, with interferon-γ signaling through JAK1/JAK2 playing a key role.3-7
- In addition to the immune elements, WNT – an important factor in melanocyte differentiation – has been implicated in the lack of re-pigmentation in vitiligo lesions.8
- Studies in alopecia have shown that – in addition to immune suppression – JAK inhibitors can be pro-melanogenic, and actively stimulate the WNT pathway.9
Clinical study data
- Ruxolitinib is the JAK inhibitor currently in the most advanced phase of development for vitiligo.
- 157 patients have been treated in a randomized, double-blind Phase 2 study examining three doses of topical ruxolitinib versus vehicle.
- At Week 24, up to 50% of ruxolitinib patients achieved a facial VASI50, compared to just 3% with vehicle. At the 52-week mark, one-third of patients on the highest dose had achieved facial VASI90; there were also good results for total VASI on other body sites.
- Ruxolitinib was well tolerated, with no clinically significant or serious treatment-emergent adverse events.