Efficacy and safety of upadacitinib in adults and adolescents for treatment of non-segmental vitiligo (Viti-Up): results of two phase 3 randomised controlled studies
Abstract
Background Non-segmental vitiligo is an autoimmune inflammatory disease characterised by loss of melanocytes leading to skin depigmentation. No systemic therapies have been approved, and additional therapeutic options are needed. Here, we report the efficacy and safety of upadacitinib, a selective once-daily Janus kinase inhibitor, in adults and adolescents with non-segmental vitiligo.
MethodsViti-Up includes two phase 3, global, multicentre, randomised, double-blind, placebo-controlled studies conducted at 138 institutions in 18 countries in Asia, Europe, North America, and South America. Eligible patients were adults and adolescents aged 12 years or older with non-segmental vitiligo involving the face and body with a Facial Vitiligo Scoring Index (F-VASI) of 0•5 or greater and Total Vitiligo Area Scoring Index (T-VASI) of 5 to less than 50. Patients were randomly assigned (2:1) to receive either upadacitinib 15 mg or placebo orally once daily. The coprimary efficacy endpoints for both studies were T-VASI 50 (≥50% reduction in T-VASI score from baseline) at week 48 and F-VASI 75 (≥75% reduction in F-VASI score from baseline) at week 48 (ie, clinically meaningful repigmentation). Efficacy endpoints were analysed in the intention-to-treat population; safety endpoints were assessed in all randomly assigned patients who received at least one dose of study drug during the 48-week double-blind period. Viti-Up is registered on ClinicalTrials.gov, NCT06118411, and is ongoing. Findings Between Dec 19, 2023, and Oct 14, 2024, 750 individuals were assessed for eligibility. 308 individuals in Viti-Up-1 (206 assigned to upadacitinib and 102 assigned to placebo; 156 [51%] female and 152 [49%] male) and 306 in Viti-Up-2 (205 assigned to upadacitinib and 101 assigned to placebo; 151 [49%] female and 155 [51%] male) were randomly assigned. At week 48, a greater proportion receiving upadacitinib versus placebo reached the coprimary endpoints of T-VASI 50 (Viti-Up-1: upadacitinib 40 [19%] participants vs placebo six [6%]; Viti-Up-2: upadacitinib 44 [21%] vs placebo six [6%]; p<0•001) and F-VASI 75 (Viti-Up-1: upadacitinib 52 [25%] vs placebo six [6%]; Viti-Up-2: upadacitinib 48 [23%] vs placebo seven [7%]; p<0•001). There were no reports of adjudicated major adverse cardiovascular events, adjudicated venous thromboembolic events, adjudicated gastrointestinal perforations, active tuberculosis, lymphoma, non-melanoma skin cancer, or opportunistic infections other than herpes zoster in either study. Safety data were consistent with the overall upadacitinib safety profile. Interpretation Upadacitinib 15 mg daily provides clinically meaningful repigmentation of both the face and total body for adults and adolescents with non-segmental vitiligo, with no new safety signals identified.
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BibTeX
@article{passeron2026efficacy,
title = {{Efficacy and safety of upadacitinib in adults and adolescents for treatment of non-segmental vitiligo (Viti-Up): results of two phase 3 randomised controlled studies}},
author = {Thierry Passeron and Vimal H Prajapati and Raja Sivamani and Khaled Ezzedine and Nanja van Geel and Iltefat Hamzavi and Amit G Pandya and David Rosmarin and Leihong Xiang and Tamio Suzuki and Seemal Desai and Mehdi Rashighi and Smitha Suravaram and Irina Fish and Xiaofei Hu and Xiaoqiang Wu and Minglu Wang and Yao Yu and Ahmed M Soliman and Heidi S Camp and Henrique D Teixeira and Stavonnie Patterson and Bethanee J Schlosser and Julien Seneschal},
journal = {The Lancet},
year = {2026},
month = sep,
doi = {10.1016/s0140-6736(26)00896-2},
url = {https://u-bordeaux.hal.science/hal-05745527v1},
note = {CC BY 4.0},
}
RIS
TY - JOUR
TI - Efficacy and safety of upadacitinib in adults and adolescents for treatment of non-segmental vitiligo (Viti-Up): results of two phase 3 randomised controlled studies
AU - Thierry Passeron
AU - Vimal H Prajapati
AU - Raja Sivamani
AU - Khaled Ezzedine
AU - Nanja van Geel
AU - Iltefat Hamzavi
AU - Amit G Pandya
AU - David Rosmarin
AU - Leihong Xiang
AU - Tamio Suzuki
AU - Seemal Desai
AU - Mehdi Rashighi
AU - Smitha Suravaram
AU - Irina Fish
AU - Xiaofei Hu
AU - Xiaoqiang Wu
AU - Minglu Wang
AU - Yao Yu
AU - Ahmed M Soliman
AU - Heidi S Camp
AU - Henrique D Teixeira
AU - Stavonnie Patterson
AU - Bethanee J Schlosser
AU - Julien Seneschal
PY - 2026
DA - 2026/09/10
JO - The Lancet
DO - 10.1016/s0140-6736(26)00896-2
UR - https://u-bordeaux.hal.science/hal-05745527v1
AB - Background Non-segmental vitiligo is an autoimmune inflammatory disease characterised by loss of melanocytes leading to skin depigmentation. No systemic therapies have been approved, and additional therapeutic options are needed. Here, we report the efficacy and safety of upadacitinib, a selective once-daily Janus kinase inhibitor, in adults and adolescents with non-segmental vitiligo. MethodsViti-Up includes two phase 3, global, multicentre, randomised, double-blind, placebo-controlled studies conducted at 138 institutions in 18 countries in Asia, Europe, North America, and South America. Eligible patients were adults and adolescents aged 12 years or older with non-segmental vitiligo involving the face and body with a Facial Vitiligo Scoring Index (F-VASI) of 0•5 or greater and Total Vitiligo Area Scoring Index (T-VASI) of 5 to less than 50. Patients were randomly assigned (2:1) to receive either upadacitinib 15 mg or placebo orally once daily. The coprimary efficacy endpoints for both studies were T-VASI 50 (≥50% reduction in T-VASI score from baseline) at week 48 and F-VASI 75 (≥75% reduction in F-VASI score from baseline) at week 48 (ie, clinically meaningful repigmentation). Efficacy endpoints were analysed in the intention-to-treat population; safety endpoints were assessed in all randomly assigned patients who received at least one dose of study drug during the 48-week double-blind period. Viti-Up is registered on ClinicalTrials.gov, NCT06118411, and is ongoing. Findings Between Dec 19, 2023, and Oct 14, 2024, 750 individuals were assessed for eligibility. 308 individuals in Viti-Up-1 (206 assigned to upadacitinib and 102 assigned to placebo; 156 [51%] female and 152 [49%] male) and 306 in Viti-Up-2 (205 assigned to upadacitinib and 101 assigned to placebo; 151 [49%] female and 155 [51%] male) were randomly assigned. At week 48, a greater proportion receiving upadacitinib versus placebo reached the coprimary endpoints of T-VASI 50 (Viti-Up-1: upadacitinib 40 [19%] participants vs placebo six [6%]; Viti-Up-2: upadacitinib 44 [21%] vs placebo six [6%]; p<0•001) and F-VASI 75 (Viti-Up-1: upadacitinib 52 [25%] vs placebo six [6%]; Viti-Up-2: upadacitinib 48 [23%] vs placebo seven [7%]; p<0•001). There were no reports of adjudicated major adverse cardiovascular events, adjudicated venous thromboembolic events, adjudicated gastrointestinal perforations, active tuberculosis, lymphoma, non-melanoma skin cancer, or opportunistic infections other than herpes zoster in either study. Safety data were consistent with the overall upadacitinib safety profile. Interpretation Upadacitinib 15 mg daily provides clinically meaningful repigmentation of both the face and total body for adults and adolescents with non-segmental vitiligo, with no new safety signals identified.
SN - 0140-6736
SN - 1474-547X
ER -