Efficacy and safety of oral ritlecitinib for the treatment of active nonsegmental vitiligo: A randomized phase 2b clinical trial

Abstract

Vitiligo is a chronic autoimmune disorder characterized by depigmented patches of the skin. To evaluate the efficacy and safety of ritlecitinib, an oral JAK3 (Janus kinase)/TEC (tyrosine kinase expressed in hepatocelluar carcinoma) inhibitor, in patients with active nonsegmental vitiligo in a phase 2b trial (NCT03715829). Patients were randomized to once-daily oral ritlecitinib ± 4-week loading dose (200/50mg, 100/50mg, 30mg, or 10mg) or placebo for 24weeks (dose-ranging period). Patients subsequently received ritlecitinib 200/50mg daily in a 24-week extension period. The primary efficacy endpoint was percent change from baseline in Facial-Vitiligo Area Scoring Index at week 24. A total of 364 patients were treated in the dose-ranging period. Significant differences from placebo in percent change from baseline in Facial-Vitiligo Area Scoring Index were observed for the ritlecitinib 50mg groups with (-21.2 vs 2.1; P<.001) or without (-18.5 vs 2.1; P<.001) a loading dose and ritlecitinib 30mg group (-14.6 vs 2.1; P=.01). Accelerated improvement was observed after treatment with ritlecitinib 200/50mg in the extension period (n=187). No dose-dependent trends in treatment-emergent or serious adverse events were observed across the 48-week treatment. Patients with stable vitiligo only were excluded. Oral ritlecitinib was effective and well tolerated over 48weeks in patients with active nonsegmental vitiligo.