FDA-Approved Ruxolitinib Cream Restores Skin Pigment in 50% of Vitiligo Patients, Finds Study

The newly approved ruxolitinib (Opzelura) cream is proving to be a game changer for millions of people living with vitiligo, a disease that causes the skin to lose its natural color. The U.S. Food and Drug Administration approved ruxolitinib for vitiligo in people aged 12 and older in July. The drug, part of a class known as Janus kinase (JAK) inhibitors, targets JAK, a molecule involved in the development and progression of vitiligo.

“This is a major milestone as ruxolitinib cream is the first treatment that’s FDA approved to re-pigment patients with vitiligo,” said study author Dr. David Rosmarin, a dermatologist and vice chair for education and research in the department of dermatology at Tufts Medical Center in Boston.

About half of people with vitiligo who used the cream got 75% or more pigment back on the face and 50% or more pigment back on their whole body after a year of use, the study found. More than one-third of adults and more than 50% of adolescents in the study said their vitiligo was no longer noticeable or a lot less noticeable after a year.

“Even patients with vitiligo for over 30 years can still improve with this treatment,” Rosmarin said.

Before the new cream’s approval, dermatologists used topical steroids, topical calcineurin inhibitors, and phototherapy to treat vitiligo, he noted. These treatments had limitations and could have side effects, such as skin lightening and thinning or burning and stinging at the application site.

Vitiligo experts welcome the new medication and are optimistic about the vitiligo treatment pipeline. Dr. Victor Huang, director of the Vitiligo Clinic at the University of California, Davis, said, “The development of this new medication is exciting to the community for the treatment it offers, the validation of the underlying science of vitiligo it represents, and for the novel treatments to come it promises.”

Dr. Iltefat Hamzavi, a dermatologist at Henry Ford Hospital in Detroit, agreed that this new cream is a big deal for people with vitiligo. “We never know how well a medication can work in a large group and also how safe it is, [and] we have that information now and that is a tremendous hurdle to overcome for a disease that had no approved treatments.”

Dr. Liv Eidsmo, a professor of translational skin and immunology at the University of Copenhagen, in Denmark, wrote an editorial accompanying the new study. Topical treatment targets the affected sites directly and lowers the risk of systemic effects, she said.

“Patients with vitiligo finally have the hope of efficient treatments, with several new immuno-modulating drugs in different phases of clinical trials,” Eidsmo added.