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Topical Cream Ruxolitinib Proven Highly Effective for Skin Re-Pigmentation in People with Vitiligo

11/15/2022

Phase III clinical trial results showed better than 75 percent improvement on face, 50 percent improvement on body for half of patients in study; ruxolitinib granted FDA approval as first-ever topical cream for skin re-pigmentation in patients with vitiligo

 

David Rosmarin, MD

An international phase III clinical trial, based on research initiated at Tufts Medical Center in Boston by David Rosmarin, MD, has found that topical cream, ruxolitinib delivered convincing results at reversing the effects of vitiligo, an autoimmune disease causing loss of skin pigmentation. The study's findings were published in The New England Journal of Medicine on October 20, 2022. Based on the results from the clinical trial, the FDA granted full approval of ruxolitinib for vitiligo patients ages 12 and older on July 18, 2022; it is the first-ever FDA-approved topical treatment for skin re-pigmentation in patients with vitiligo.

The one-year, international phase III clinical trial enrolled 343 patients at 70 sites, primarily in North America and Europe. About half of study participants who were randomized to receive topical ruxolitinib experienced at least a 75 percent re-pigmentation improvement on their faces and better than 50 percent re-pigmentation of their bodies. Topical ruxolitinib also showed an excellent safety profile, with only mild side effects in some patients, including redness and irritation at the application site and mild acne.

"It is both satisfying and gratifying to reach this point where people with vitiligo from around the world can access this treatment to re-pigment their skin, if they so choose," said Dr. Rosmarin, Dermatologist and Vice Chair for Education and Research in the Department of Dermatology at Tufts Medical Center, who has been leading the research on ruxolitinib as a treatment for vitiligo since 2014. "Alternative therapies can be expensive and burdensome, with limited efficacy. Ruxolitinib is simple to use and effective, and now with FDA-approval, I look forward to it becoming widely available for those who want and need it."

There is no known cause of vitiligo, a skin pigmentation disease in which an overactive immune system attacks pigment cells in the body, causing the skin to lose its natural color. Vitiligo may range from mild to severe and can be found anywhere on the body, although it typically affects exposed areas, such as the face and hands. While vitiligo is not evident at birth, about half of vitiligo patients contract the disease before age 20. Approximately 70 million people – about one percent of the worldwide population -- are affected by vitiligo, including late musical artist Michael Jackson, actor John Hamm, model Winnie Harlow, retired basketball star Rasheed Wallace and comedian Steve Martin. While vitiligo affects all ethnic groups equally, it is more visibly evident in ethnic minorities with darker skin.

"Topical ruxolitinib has the potential to be life-changing for millions of patients with vitiligo and change the standard of care for how physicians approach treating this disease," said Dr. Rosmarin. "There is still a lot more work to be done, but it is a milestone step forward to have an effective, accessible re-pigmentation option for this patient population."

The study was funded by biopharmaceutical research company, Incyte Corporation, which produces ruxolitinib.