Safety and Efficacy of Ruxolitinib Cream for the Treatment of Vitiligo: 104-Week Data From a Phase 2 Study

Vitiligo is a chronic autoimmune disease resulting in skin depigmentation. Ruxolitinib cream, a Janus kinase (JAK) 1/JAK2 inhibitor, demonstrated significant repigmentation in a phase 2, dose- ranging, randomized study of adult patients with vitiligo (N=157) when applied to ≤20% total body surface area. Of 80 patients who applied 0.5% ruxolitinib cream once daily (QD), 1.5% QD, or 1.5% twice daily (BID) and completed the 52-week randomized period, 77 continued to apply 1.5% ruxolitinib cream BID in the open-label period for another 52 weeks. Twenty-seven patients (35.1%) discontinued open-label treatment, most commonly because of withdrawal by patient (n=18). No significant safety signals were observed during the 52-week open-label period; 7 patients (9.1%) had a treatment-related adverse event (TRAE; all grade 1/2). Among patients initially randomized to receive 1.5% ruxolitinib cream BID (n=33), TRAEs were reported in 11 patients (33.3%; all grade 1 [n=10] or 2 [n=1]) over the 104-week study period. Regardless of dose at initial randomization, no accumulation of AEs or clinically relevant changes in hemoglobin or platelet levels were observed over the 104-week treatment period. Among all evaluable patients with a Week 104 study visit (n=55), F-VASI50, F-VASI75, and F-VASI90 were achieved by 83.6%, 65.5%, and 52.7% of patients, respectively; T-VASI50 was achieved by 58.2% of patients. In summary, treatment with ruxolitinib cream was well tolerated over a 2-year period and resulted in substantial repigmentation of vitiligo lesions.