The FDA has approved the topical Janus kinase inhibitor Opzelura for the treatment of nonsegmental vitiligo in adolescents and adults, Incyte announced in a press release.
Opzelura (ruxolitinib) cream 1.5% is the first FDA-approved treatment for repigmentation in patients with vitiligo.
“This is the start of offering some hope and options to patients who want to be repigmented and hopefully give them the chance to do that,” David Rosmarin, MD, dermatologist and vice chair of research and education for the department of dermatology at Tufts Medical Center and a lead investigator of the ruxolitinib trials, told Healio.
David Rosmarin
The approval was based on data from two phase 3 TRuE-V clinical trials, which included more than 600 people with nonsegmental vitiligo.
At week 24, approximately 30% of patients treated with Opzelura had at least a 75% improvement in facial Vitiligo Area Scoring Index (F-VASI75), compared with approximately 8% and 13% of those in the vehicle arms.
At week 52, approximately 50% of patients achieved F-VASI75 and about 30% achieved F-VASI90.
“It’s important to know that repigmentation takes time. Some patients respond early, some may take a little more time. But the longer you use the medicine, the more benefits the patient will get,” Rosmarin said. “When we look at different subgroups … all the patients seem to have a consistent response.”
The drug’s side effects included mild acne and application site reaction.
“With the approval of Opzelura in nonsegmental vitiligo, Incyte has once again delivered a treatment to patients with high unmet medical need who previously had no approved therapies,” Hervé Hoppenot, Incyte CEO, said in the release. “We are proud of Incyte’s scientists and development teams that have made this milestone possible, and we’re pleased that eligible vitiligo patients now have a choice to address repigmentation.”
Opzelura was previously approved for the short-term, noncontinuous treatment of mild to moderate dermatitis.
Perspective
This is a momentous occasion, one for which we have been waiting for thousands of years — literally. Topical ruxolitinib, or Opzelura, has just been approved for the treatment of vitiligo by the FDA.
I started writing about this drug in 2015, when we were the first to give it to a vitiligo patient orally and it worked. In 2017, Dr. David Rosmarin, of Tufts Medical Center, had ruxolitinib put into a cream and showed it worked topically for patients with vitiligo on their face.
While Opzelura was already approved in September 2021 for atopic dermatitis, most couldn’t get it covered by insurance for vitiligo because it wasn’t approved for it. But now it is! The phase 3 clinical trial enrolled volunteers aged as young as 12 years, and so the approval covers its use for children 12 years and older. Safety data from the trial looked good. The only side effect noted in those using the drug more than placebo, and therefore attributable to the drug, was localized acne bumps in about 10% to 15% of users.
However, please note — Opzelura has a “black box warning” on the label, meaning that the FDA wants you to know that there could be a small risk for serious infections, heart disease, blood clotting, cancer or even death. These were not seen in the clinical trials for the topical drug ruxolitinib but have been reported in small numbers of those taking the oral version of the drug, which results in very large concentrations in the blood. These concentrations were not seen in trials when volunteers used the cream, but this is a “better safe than sorry” approach by the FDA, so that users are made fully aware.I’m really excited about this and expect this is only the tip of the iceberg. There are so many more ideas being tested now, and I anticipate more options will be available for patients soon.
John E Harris, MD, PhD
Professor and Chair, Department of Dermatology
Founding Director, Vitiligo Clinical Research Center
Founding Director, Autoimmune Therapeutics Institute
UMass Chan Medical School
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