Topical Ruxolitinib Promising for Nonsegmental Vitiligo in Phase 3 Trials

The efficacy and safety of ruxolitinib cream for the treatment of adults and adolescents with nonsegmental vitiligo is assessed.

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Ruxolitinib cream was clinically superior to vehicle in patients with nonsegmental vitiligo in a pair of phase 3 studies, according to study results presented at the 2022 Annual Meeting of the American Academy of Dermatology (AAD), held from March 25 to 29, 2022 in Boston, Massachusetts.

Investigators assessed the efficacy and safety of ruxolitinib cream after 24 weeks of double-blind treatment using pooled data from the phase 3 TRuE-V1 and TRuE-V2 studies of adults and adolescents with nonsegmental vitiligo.

Eligible participants were aged 12 years or older and diagnosed with nonsegmental vitiligo and depigmented areas covering 10% or less of total body surface area (BSA), including 0.5% or greater BSA on the face and 3% or less BSA on nonfacial areas, scores 0.5 or greater on the facial Vitiligo Area Scoring Index (F-VASI), and scores 3 or higher on the total VASI (T-VASI).

A total of 674 patients were randomly assigned in the 2 trials (ruxolitinib cream, n=450; vehicle, n=224). Overall, participants had a mean (SD) age of 39.6 (15.1) years, 53.1% were woman, and 81.9% were White. Also, 67.5% of patients had skin phototypes III to VI, and the baseline mean F-VASI and T-VASI values were 0.92 and 6.69, respectively.

The primary endpoint was the percentage of patients who achieved 75% or greater improvement at week 24 from baseline in F-VASI (F-VASI75).

At week 24, a significantly greater percentage of patients who used ruxolitinib cream achieved F-VASI75 vs those who received vehicle (30.1% vs 10.8%; P <.0001). The percentage of patients with F-VASI50 and F-VASI90 responses was significantly higher with ruxolitinib cream vs those with vehicle (both P ≤.0001). A significantly greater percentage of patients who used ruxolitinib cream had T-VASI50 at week 24 vs those with vehicle (23.2% vs 8.1%; P <.0001).

The percentage of patients who achieved a response on the Vitiligo Noticeability Scale at week 24 with ruxolitinib cream vs vehicle was 23.2% vs 5.3%, respectively (P <.0001). The least squares mean percentage change from baseline in F-BSA was –22.8 with ruxolitinib cream compared with –3.1 for vehicle (P <.0001) at week 24.

Visible improvement was observed in repigmentation of facial and nonfacial lesions in patients who used ruxolitinib cream, the researchers noted.

Treatment-emergent adverse events (TEAEs) considered to be treatment related were mild or moderate in severity and occurred in 14.7% of patients who used ruxolitinib cream and in 7.6% who used vehicle. Application site acne (4.9%) and pruritus (4.7%) were the most common treatment-related TEAEs in those who used ruxolitinib cream. Serious TEAEs occurred in 1.8% of participants who used ruxolitinib cream and in 0.4% of those who received vehicle.

“Ruxolitinib cream demonstrated clinically meaningful superiority to vehicle for the primary and all key secondary endpoints in the pooled analysis of the TRuE-V1/TRuE-V2 phase 3 studies, confirming phase 2 findings,” concluded the investigators.

Disclosure: This study was funded by Incyte Corporation. Some of the study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of disclosures.


Rosmarin D, Ezzedine K, Desai SR, et al. Efficacy and safety of ruxolitinib cream for the treatment of vitiligo: week 24 pooled analysis of the TRuE-V phase 3 studies. Presented at: American Academy of Dermatology (AAD) 2022 Annual Meeting; March 25-29, 2022. Poster 34789.

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