A Review on the Use of Topical Ruxolitinib for the Treatment of Vitiligo

July 2023 | Volume 22 | Issue 7 | 664 | Copyright © July 2023


Published online June 27, 2023

doi:10.36849/JDD.7268.

Meghan C Grossmann BSa, Wasim Haidari MDa, Steven R. Feldman MD PhDa,b,c,d

aCenter for Dermatology Research, Department of Dermatology, Wake Forest School of Medicine, Winston-Salem, NC
bDepartment of Pathology, Wake Forest School of Medicine, Winston-Salem, NC
cDepartment of Social Sciences & Health Policy, Wake Forest School of Medicine, Winston-Salem, NC
dDepartment of Dermatology, University of Southern Denmark, Odense, Denmark

Abstract
Background: This article describes the clinical trial, safety, and efficacy of ruxolitinib 1.5% cream or repigmentation in patients with vitiligo.
Data Sources: A systematic review was done using ruxolitinib or Opzelura in MEDLINE (PubMed) and EMBASE. ClinicalTrials.gov was used to identify ongoing or unpublished studies.
Study Selection and Data Extraction: Studies included were written in English and relevant to pharmacology, clinical trials, safety, and efficacy.
Data Synthesis: In two 52-week phase 3 trials, 52.0% of subjects had at least 75% improvement in their Facial Vitiligo Area Scoring Index (F-VASI).
Relevance to Patient Care and Clinical Practice: Ruxolitinib is a topical Janus kinase (JAK) inhibitor newly approved by the US Food and Drug Administration for repigmentation in patients with vitiligo.
Conclusion: Topical ruxolitinib is the first medication approved for repigmentation in patients with vitiligo. It is a safe and effective treatment; however, cost may be a barrier to some patients when prescribing this medication. Trials to compare the efficacy and side effect profile of topical ruxolitinib with other topical treatments are still needed.

Grossmann MC, Haidari W, Feldman SR. A Review on the use of topical ruxolitinib for the treatment of vitiligo. J Drugs Dermatol. 2023;22(7):664-667. doi:10.36849/JDD.7268.

INTRODUCTION

Vitiligo is a multifactorial depigmentation disorder characterized by the destruction of melanocytes, resulting in loss of pigmentation of the skin.1 An autoimmune process plays an important role in the disease pathogenesis, with CD8+ T cells in vitiligo lesions, producing a variety of cytokines, such as interferon‐gamma (IFN‐γ).2 Treatment up until now has been with the use of topical corticosteroids, immunomodulators, and narrow band-ultraviolet (UV) B, none of which is approved by the US Food and Drug Administration (FDA) for the treatment of vitiligo.1 Monobenzone is an FDA-approved treatment for the depigmentation of vitiligo, offering patients with more extensive disease an option for treatment by inducing melanocyte necrosis giving patients a more uniform skin tone.3

Ruxolitinib 1.5% cream, a topical Janus kinase (JAK) inhibitor, was approved by the FDA in July 2022 for treatment of nonsegmental vitiligo in patients ages 12 and up.4 Topical ruxolitinib is the first drug FDA-approved for repigmentation in patients with vitiligo.4 Ruxolitinib can be used topically up to 60 grams in one week, or 100 grams over 2 weeks on ≤10% body surface area (BSA).5 The purpose of this review is to describe the pharmacology, clinical trials, safety, and efficacy of ruxolitinib in the treatment of vitiligo.

MATERIALS AND METHODS

A systematic review was performed using the terms ruxolitinib OR opzelura in MEDLINE (PubMed) and EMBASE databases. Available studies were considered for inclusion if they were written in English and related to pharmacology, clinical trials, adverse events (AEs), and safety prior to July 2022. References of the included sources were also searched to identify additional studies for inclusion. ClinicalTrials.gov was searched to identify ongoing or unpublished studies.

RESULTS

Drug Pharmacology
Mechanism of Action
There is an ongoing clinical trial (TRuE-MOA NCT04896385) to assess the mechanism of action of topical ruxolitinib.6 Ruxolitinib is a selective JAK1 and JAK2 inhibitor that works on multiple cytokines and growth factors. The therapeutic relevance of JAK enzyme inhibition is not currently known.7