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

J Am Acad Dermatol. 2023 Feb;88(2):395-403. doi: 10.1016/j.jaad.2022.11.005. Epub 2022 Nov 9.


Background: Vitiligo is a chronic autoimmune disorder characterized by depigmented patches of the skin.

Objective: 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).

Methods: Patients were randomized to once-daily oral ritlecitinib ± 4-week loading dose (200/50 mg, 100/50 mg, 30 mg, or 10 mg) or placebo for 24 weeks (dose-ranging period). Patients subsequently received ritlecitinib 200/50 mg 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.

Results: 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 50 mg groups with (-21.2 vs 2.1; P < .001) or without (-18.5 vs 2.1; P < .001) a loading dose and ritlecitinib 30 mg group (-14.6 vs 2.1; P = .01). Accelerated improvement was observed after treatment with ritlecitinib 200/50 mg in the extension period (n = 187). No dose-dependent trends in treatment-emergent or serious adverse events were observed across the 48-week treatment.

Limitations: Patients with stable vitiligo only were excluded.

Conclusions: Oral ritlecitinib was effective and well tolerated over 48 weeks in patients with active nonsegmental vitiligo.

Keywords: JAK inhibitor; JAK/STAT signaling; TEC inhibitor; VASI; randomized clinical trial; ritlecitinib; skin depigmentation; vitiligo.

Publication types

  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Chronic Disease
  • Double-Blind Method
  • Humans
  • Janus Kinases
  • Protein Kinase Inhibitors / adverse effects
  • Skin / pathology
  • Treatment Outcome
  • Vitiligo* / drug therapy
  • Vitiligo* / pathology


  • Janus Kinases
  • Protein Kinase Inhibitors

Associated data

  • ClinicalTrials.gov/NCT03715829