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. 2025 Oct 22.
doi: 10.1007/s13555-025-01555-3. Online ahead of print.

Long-Term Integrated Safety Summary of Ruxolitinib Cream in Phase 3 Clinical Trials of Patients with Vitiligo

David Rosmarin  1 , Amit G Pandya  2   3 , Thierry Passeron  4   5 , Seth B Forman  6 , Jacek Zdybski  7 , Mark Amster  8 , Christina Feser  9 , Kim A Papp  10   11 , Anthony Nuara  12 , Deanna Kornacki  13 , Shaoceng Wei  13 , Haobo Ren  13 , John E Harris  14 , Khaled Ezzedine  15
Affiliations

Long-Term Integrated Safety Summary of Ruxolitinib Cream in Phase 3 Clinical Trials of Patients with Vitiligo

David Rosmarin et al. Dermatol Ther (Heidelb). .

Abstract

Introduction: Ruxolitinib cream demonstrated superior repigmentation versus vehicle at week 24 with continued improvement through week 104 in phase 3 studies of patients (aged ≥ 12 years) with nonsegmental vitiligo. Here, we evaluated long-term safety of ruxolitinib cream in an integrated analysis of phase 3 vitiligo studies.

Methods: Reported incidence and exposure-adjusted incidence rates (EAIRs) of treatment-emergent adverse events (TEAEs) were determined.

Results: Over 104 weeks, 673 patients with vitiligo applied either ruxolitinib cream (n = 637; 867.9 person-years [PY]) or vehicle (n = 270; 131.1 PY). TEAEs (EAIR, patients/100 PY) with ruxolitinib cream versus vehicle occurred in 62.6% (46.0) versus 37.0% (76.3) of patients, most commonly nasopharyngitis (7.2% [5.3] vs 2.6% [5.3]) and application site acne (6.0% [4.4] vs 1.1% [2.3]). No serious treatment-related TEAEs were reported with ruxolitinib cream. EAIRs (patients/100 PY) were low for acne-related TEAEs (7.1), skin and subcutaneous tissue infections (4.0), cytopenias (2.4), and liver enzyme elevations (2.2). Malignancies, serious infections, and thromboembolic events were rare (0.7, 0.5, and 0.2 patients/100 PY, respectively), and none were considered related to treatment. No major adverse cardiovascular events or deaths occurred.

Conclusion: Ruxolitinib cream demonstrated tolerability, with no unexpected safety findings through 2 years in patients with vitiligo. Graphical Plain Language Summary available for this article.

Trial registration: Clinicaltrials.gov identifiers, NCT04052425 (registered on August 8, 2019), NCT04057573 (registered on August 14, 2019), and NCT04530344 (registered on August 25, 2020).

Keywords: Janus kinase inhibitor; Ruxolitinib cream; Vitiligo.

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Conflict of interest statement

Declarations. Conflict of Interest: David Rosmarin is an Editorial Board member of Dermatology and Therapy and was not involved in the selection of peer reviewers for the manuscript nor any of the subsequent editorial decisions; has served as a consultant, speaker, or investigator for AbbVie, Abcuro, Almirall, AltruBio, Amgen, Arena, Astria, Boehringer Ingelheim, Bristol Myers Squibb, Celgene, Concert, CSL Behring, Dermavant Sciences, Dermira, Dualitas, EMD Serono, Galderma, Incyte Corporation, Janssen, Kymera, Kyowa Kirin, Lilly, Merck, Nektar, Novartis, Pfizer, RAPT Therapeutics, Recludix, Regeneron Pharmaceuticals, Revolo Biotherapeutics, Sanofi, Sun Pharmaceuticals, UCB, Viela Bio, and Zura Bio. Amit G. Pandya has served as an investigator for Aclaris Therapeutics, Immune Tolerance Network, Incyte Corporation, and Pfizer; a consultant for AbbVie, Arcutis, Avita Medical, Chromaderm, Immune Tolerance Network, Incyte Corporation, Pfizer, TWi, Viela Bio, and Villaris; and holds stock options for Tara Medical and Zerigo Health. Thierry Passeron has received grants and/or honoraria from AbbVie, ACM Pharma, Almirall, Amgen, Astellas, Bristol Myers Squibb, Calypso, Celgene, Eli Lilly, Galderma, Genzyme/Sanofi, GlaxoSmithKline, Incyte Corporation, Janssen, LEO Pharma, Novartis, Pfizer, Sun Pharmaceuticals, Takeda, UCB, and Vyne Therapeutics; is the cofounder of NIKAIA Pharmaceuticals; and has patents on WNT agonists and GSK3b antagonists for repigmentation of vitiligo and on the use of CXCR3B blockers in vitiligo. Seth B. Forman has received honoraria, clinical research grants, or fees as a consultant, speaker, advisory board member, and/or investigator for AbbVie, Aclaris Therapeutics, Asana BioSciences, AstraZeneca, Athenex, Celgene Corporation, Cutanea Life Sciences, Eli Lilly, Incyte Corporation, Innovaderm Research, Novartis, Pfizer, Promius Pharma, Regeneron, UCB, Valeant Pharmaceuticals North America, and Xbiotech. Jacek Zdybski has served as an investigator for Almirall, Amgen, Bristol Myers Squibb, Celgene, Eli Lilly, Galderma, Incyte Corporation, Innovaderm, Pfizer, Regeneron, Sun Pharmaceuticals, and Syneos Health. Mark Amster has been a primary investigator and received revenue from Acrotech Biopharma, Amgen, Arcutis Biotherapeutics, Bausch Health, Biopharmex, Boehringer Ingelheim, Botanix Pharmaceuticals, Cara Therapeutics, Concert Pharmaceuticals, Dermavant, Dermira, DermTech, Dr. Reddys Laboratories, Eli Lilly, Foamix, Highlight Pharmaceuticals, Incyte Corporation, Janssen, Kademis Ltd, LEO Pharma, Mayne Pharma, Mitsubishi Tanabe Pharma, Novartis, Novella, Pfizer, RAPT Therapeutics, Trevi Therapeutics, UCB Biopharma, Ventyx Biosciences, Vitae Pharmaceuticals, and Xbiotech USA and has received honoraria as a consultant for AbbVie, Cara Pharmaceuticals, Incyte Corporation, and Janssen. Christina Feser has served as a speaker, consultant, advisory board member, and/or investigator for AbbVie, Aclaris, Acrotech Biopharma, Almirall, Amgen, Arcutis, Bristol Myers Squibb, Castle Biosciences, Cutanea, Eli Lilly and Company, Incyte Corporation, Kymab, LEO Pharma, Merck, MoonLake, Nektar Therapeutics, Novan, Novartis, Paradigm Medical, ParaPro, Pfizer, Skin Life Science, SkinTech, Takeda, Technoderma, and Ventyx. Kim A. Papp has received honoraria and/or research grants from AbbVie, Acelyrin, Akros, Alumis, Amgen, Arcutis, Bausch Health/Valeant, Boehringer Ingelheim, Bristol Myers Squibb, Can-Fite Biopharma, Celltrion, Concert Pharmaceuticals, Dermavant, Dermira, DiCE Pharmaceuticals, DiCE Therapeutics, Eli Lilly, Evelo Biosciences, Forbion, Galderma, Horizon Therapeutics, Incyte Corporation, Janssen, Kymab, Kyowa Hakko Kirin, LEO Pharma, Meiji Seika Pharma, Mitsubishi Pharma, Nimbus Therapeutics, Novartis, Pfizer, Reistone, Sandoz, Sanofi-Aventis/Genzyme, Sun Pharmaceuticals, Takeda, Tarsus Pharmaceuticals, UCB Pharma, and Zai Lab. Anthony Nuara has received research funding from AbbVie, Aclaris Therapeutics, AOBiome, AstraZeneca, Boehringer Ingelheim, Galderma, Incyte Corporation, Novartis, and Pfizer and has served as a paid speaker and/or consultant for AbbVie, Amgen, Castle Biosciences, Bristol Myers Squibb, Dermavant, Galderma, Incyte Corporation, Janssen, and Novartis. Deanna Kornacki, Shaoceng Wei, and Haobo Ren are employees and shareholders of Incyte Corporation. John E. Harris has served as a consultant for AbbVie, Aclaris Therapeutics, BiologicsMD, EMD Serono, Genzyme/Sanofi, Janssen, Pfizer, Rheos Medicines, Sun Pharmaceuticals, TeVido BioDevices, The Expert Institute, 3rd Rock Ventures, and Villaris Therapeutics; has served as an investigator for Aclaris Therapeutics, Celgene, Dermira, EMD Serono, Genzyme/Sanofi, Incyte Corporation, LEO Pharma, Pfizer, Rheos Medicines, Stiefel/GlaxoSmithKline, Sun Pharmaceuticals, TeVido BioDevices, and Villaris Therapeutics; holds equity in Aldena Therapeutics, NIRA Biosciences, Rheos Medicines, TeVido BioDevices, and Villaris Therapeutics; is a scientific founder of Aldena Therapeutics, NIRA Biosciences, and Villaris Therapeutics; and has patents pending for IL-15 blockade for treatment of vitiligo, JAK inhibition with light therapy for vitiligo, and CXCR3 antibody depletion for treatment of vitiligo. Khaled Ezzedine has served as a consultant for AbbVie, Almirall, Bristol Myers Squibb, Incyte Corporation, La Roche-Posay, Lilly, Pfizer, Pierre Fabre Pharmaceuticals, and Sanofi. Ethical Approval: These studies were conducted in accordance with Good Clinical Practice guidelines, provisions of the Declaration of Helsinki, and applicable regulations. Study protocols were approved by each site’s institutional review board.

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