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New long-term extension data from the pivotal TRuE-V program show treatment continuation, as well as post-relapse re-initiation, is efficacious and safe in patients.
Approximately 4 in 10 of patients who withdrew from ruxolitinib cream (Opzelura) treatment after achieving a significant facial repigmentation response had maintained that response after 1 year post-treatment, according to new long-term extension findings.1
In late-breaking data presented at the American Academy of Dermatology (AAD) 2023 Annual Meeting in New Orleans this weekend, a team of US investigators reported that patients who achieved facial clearance per Vitiligo Area Severity Index scores of ≥75% (F-VASI75) in the drug’s pivotal phase 3 were more likely to maintain F-VASI75 than they were to relapse 1 year after treatment withdrawal.
The findings, presented by investigator John Harris, MD, PhD, dermatology chair at UMass Chan School of Medicine, additionally provided critical data as to how the lone drug approved by the US Food and Drug Administration (FDA) for vitiligo can be reinitiated in relapsed patients, as well as its favorable safety and tolerability profile through 2 years of treatment.
The FDA approved ruxolitinib cream for the treatment of vitiligo in patients ≥12 years old in July 2022, on the basis of findings from the 52-week, phase 3 TRuE-V clinical trial program data showing approximately 30% of patients receiving ruxolitinib cream achieved F-VASI90 by week 52.2
“Now the question really is once you stop that drug, what’s the rate of relapse? How frequently will patients see vitiligo return?” Harris said in the presentation. “Interestingly, it comes back in the same place as before…but now that we have a new treatment in vitiligo, a JAK inhibitor, will it follow those same rules?”
Harris and colleagues sought to evaluate time to relapse per F-VASI <75 among adolescent and adult patients with nonsegmental vitiligo who achieved F-VASI90 in the parent TRuE-V trials and were then randomized to withdrawal in the long-term extension assessment. They additionally evaluated duration of maintained F-VASI90 response among the patients who were randomized to either withdrawal or continued treatment in the extension.
As such, the trial consisted of 2 cohorts: patients with F-VASI response ≥90 versus those with a <90 response. The first cohort was randomized to either continued 1.5% ruxolitinib cream twice-daily or withdrawal; the second cohort continued therapy. Treatment extension lasted 52 weeks, with a 30-day follow-up period.
The first cohort consisted of 116 patients randomized to either continued ruxolitinib cream (n = 58) or withdrawal (n = 58). Mean patient age was 42.0 years old; 64 (55.2%) were female and 90 (77.8%) were White.
Among patients with F-VASI ≥90 at the end of TRuE-V, a response of F-VASI ≥75 was maintained among 39.3% of patients in the withdrawal arm. Another 28.6% of patients in the withdrawal arm reached relapse, per F-VASI <75. Half of all relapse events occurred in patients in ≤4 months, and 23.2% of patients in the withdrawal arm discontinued their treatment prior to 1 year.
Another 61.8% of patients in the first cohort receiving ruxolitinib cream were able to maintain a F-VASI response of ≥90; 21.4% of patients who had withdrawn therapy maintained this benefit as well.
Among withdrawal patients in the first cohort who relapsed, a median F-VASI response of ≥75 was regained within a median 12 weeks; 12 (75.0%) of patients who relapsed during their withdrawal in the extension achieved this response level again.
“If your patients lose their pigment after withdrawing, (put them back on) topical ruxolitinib and they’ll gain it again,” Harris said.
Regarding safety, investigators reported good tolerability through 2 years of treatment; 35 (43.2%) of patients receiving ruxolitinib cream reported a treatment-emergent adverse event (TEAE) including application site dermatitis or rash, and hyperlipidemia. Only COVID-19 (n = 10 [12.3%]) occurred in ≥10% of patients.
The team concluded that many patients who first achieved a great extent of facial repigmentation with initial ruxolitinib cream were able to maintain that benefit for 1 year after withdrawal. Relapse was commonly resolved in patients who withdrew from treatment, and ruxolitinib cream was associated with no serious TEAEs through 2 years of application.
“If you stop the drug, just like everything else, vitiligo tends to come back—but not in everybody,” Harris said. “There’s a population who will maintain their repigmentation even off the drug. But if you restart them, they’ll regain.”