Findings indicate that modulation of inflammatory pathways could provide a systemic option for visible re-pigmentation in non-segmental vitiligo.
AbbVie has announced positive topline results from two replicate phase 3 pivotal studies evaluating the Janus kinase (JAK) inhibitor, upadacitinib (Rinvoq®; 15 mg once daily) in adults and adolescents aged ≥12 years with non-segmental vitiligo (NSV), the most common form of vitiligo, which affects around 80–90% of all of individuals living with the condition.1,2
NSV is characterised by symmetrical, bilateral depigmented patches on the skin, often with significant psychosocial impact.
Study design and methodology
The upadacitinib M19-044 programme was conducted under a single protocol encompassing two independent phase 3 studies (Study 1 and Study 2; NCT06118411) with separate randomization, data collection and analysis. Both trials assessed the efficacy, safety and tolerability of upadacitinib in adult and adolescent patients living with NSV who were eligible for systemic therapy.
Participants were randomised 2:1 to receive upadacitinib 15 mg once daily or placebo for 48 weeks (Period A), followed by a 112-week open-label extension phase (Period B) in which all patients received upadacitinib. In total, 614 participants with NSV were enrolled across 90 global sites.
The co-primary endpoints were:
- Achievement of Total Vitiligo Area Scoring Index 50 (T-VASI 50), defined as at least a 50% reduction from baseline in T-VASI 50 at week 48.
- Achievement of Facial Vitiligo Area Scoring Index 75 (F-VASI 75), defined as a 75% reduction from baseline in F-VASI 75 at week 48, comparing upadacitinib with placebo.
Secondary endpoints included F-VASI 50 at week 48 and F-VASI 75 at week 24 to assess the degree and timing of facial re-pigmentation, which is a key area of psychosocial importance.
Efficacy findings
Across the two phase 3 studies, treatment with upadacitinib 15 mg demonstrated markedly greater efficacy than placebo. At week 48, significantly more patients receiving upadacitinib achieved the co-primary endpoints of T-VASI 50 (19.4% and 21.5% vs 5.9% for placebo in both studies) and F-VASI 75 (25.2% and 23.4% vs 5.9% and 6.9%, respectively).
Improvements were also observed in the secondary endpoints, with F-VASI 50 responses at week 48 achieved by 48.1% and 43.4% of upadacitinib-treated patients, compared with 12.7% and 12.9% in the placebo groups.
Together, these data indicate that once-daily upadacitinib 15 mg significantly improved both total body and facial repigmentation compared with placebo after 48 weeks of treatment.
Safety findings
Across both studies, upadacitinib demonstrated a safety profile consistent with that observed in its approved indications. The most frequent treatment-emergent adverse events (TEAEs) were upper respiratory tract infection, acne and nasopharyngitis. Serious adverse events occurred in 3.9–4% of patients in Study 1 and 1–2% in Study 2, comparable between treatment and placebo groups. No new safety signals, deaths, or adjudicated cases of major cardiovascular events (MACE) or venous thromboembolism (VTE) were reported.
Clinical significance
Commenting on the results, Prof. Thierry Passeron, Chair of Dermatology at Université Côte d’Azur, said: “For many people living with vitiligo, the journey is marked by uncertainty and frustration. These results indicate that targeting the underlying inflammation may offer a systemic treatment option capable of achieving visible re-pigmentation.”
“Vitiligo is more than a skin condition, it is a chronic autoimmune disease that can profoundly affect a person’s confidence, identity and daily life,” added Kori Wallace, MD, PhD, Vice President and Global Head of Immunology Clinical Development at AbbVie. “There are currently no approved systemic medical therapies for achieving re-pigmentation in vitiligo. These phase 3 results represent a significant milestone in AbbVie’s commitment to supporting patients and expanding our immunology portfolio to deliver innovative solutions.”
References
- AbbVie announces positive topline results from phase 3 pivotal studies evaluating upadacitinib (RINVOQ) in adults and adolescents with vitiligo. Press release. AbbVie. October 29, 2025. Accessed October 29, 2025 at https://news.abbvie.com/2025-10-29-AbbVie-Announces-Positive-Topline-Results-from-Phase-3-Pivotal-Studies-Evaluating-Upadacitinib-RINVOQ-R-in-Adults-and-Adolescents-with-Vitiligo
- AL-smadi K, Imran M, Leite-Silva VR, Mohammed Y. Vitiligo: A Review of Aetiology, Pathogenesis, Treatment, and Psychosocial Impact. Cosmetics. 2023; 10(3):84. https://doi.org/10.3390/cosmetics10030084
Disclosure: This article was created by the touchDERMATOLOGY team utilizing AI as an editorial tool (ChatGPT (GPT-4o) [Large language model]. https://chat.openai.com/chat.) The content was developed and edited by human editors. No funding was received in the publication of this article.
Citation: Upadacitinib achieves significant repigmentation in phase 3 vitiligo trials. TouchDERMATOLOGY. 13 November 2025.
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