Ruxolitinib cream 1.5% produces ‘meaningful’ outcomes in vitiligo
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Ruxolitinib cream 1.5% was associated with a significant improvement compared with vehicle in two trials of patients with vitiligo, according to a study.
“Vitiligo is an autoimmune disease where interferon gamma is signaling through the Janus kinase (JAK) signaling pathway leading to the death of pigment cells melanocytes,” David Rosmarin, MD, dermatologist and vice chair of education and research for the department of dermatology at Tufts Medical Center, told Healio. “Ruxolitinib cream (Opzelura, Incyte Dermatology) is a JAK inhibitor and interferes with the pathology of the disease by blocking interferon gamma from signaling.”
The study reports on Topical Ruxolitinib Evaluation in Vitiligo Study 1 (TRuE-V1) and TRuE-V2, two phase 3, double-blind, vehicle-controlled studies of the cream conducted in North America and Europe.
“Ruxolitinib cream has gone through a number of different studies,” Rosmarin said, noting that the drug moved with effectiveness through early phase trials and produced “meaningful results” for patients.
The analysis included 674 patients aged 12 years or older, with 330 patients from TRuE-V1 and 344 from TRuE-V2.
Eligibility criteria included non-segmental vitiligo with depigmentation covering 10% or less of total body surface area, according to the study.
Participants were randomly assigned 2:1 to 1.5% ruxolitinib cream or vehicle twice daily for 24 weeks. All areas impacted by vitiligo on the face and body were to undergo treatment.
After the initial 24-week period, all patients were open to use the study drug through 52 weeks.
Improvement of at least 75% from baseline as assessed by the Vitiligo Area Scoring Index (F-VASI75) response at week 24 served as the primary outcome measure.
Results for TRuE-V1 showed that 29.8% of participants in the ruxolitinib cream 1.5% group reached the primary endpoint, compared with just 7.4% in the vehicle group (RR = 4; 95% CI, 1.9-8.4).
For TRuE-V2, 30.9% of participants in the active therapy arm reached the primary endpoint, compared with 11.4% in the vehicle arm (RR = 2.7; 95% CI, 1.5-4.9).
“The results are remarkably consistent between the phase 2 and two phase 3 studies,” Rosmarin said.
The researchers also noted that ruxolitinib cream 1.5% was superior to vehicle for key secondary endpoints, such as Vitiligo Noticeability Scale.
“Also, on subgroup analysis, whether patients had vitiligo for a few months or decades, we are still seeing a similar response in F-VASI improvement,” Rosmarin added. “In addition, whether patients had progressive or stable disease, they responded similarly.”
That said, anatomic location of vitiligo “does matter,” according to Rosmarin.
“We are seeing very good responses on the head and neck, trunk, extremities, but it is harder to treat the hands and feet,” he said.
Looking at the results through the 52-week treatment period, adverse event rates were 54.8% among patients in TRuE-V1 and 62.3% for TRuE-V2.
Acne at the application site was the most common adverse event in both trials (6.3% and 6.6%, respectively), whereas nasopharyngitis was the second most common and occurred in 5.4% of TRuE-V1 participants and 6.1% of TRuE-V2 participants. Application-site pruritis also was observed in 5.4% of patients in the first trial and 5.3% in the second trial.
“This is a major milestone as the first treatment to be FDA approved to repigment patients with vitiligo,” Rosmarin said. “It offers hope to those who want to repigment. It also paves the way for other vitiligo treatments to follow a similar pathway of trial design and for regulatory approval.”