FDA Approves First At-Home Topical Treatment for Vitiligo

For the first time, people with the skin discoloration disorder vitiligo will have a topical treatment.

The U.S. Food and Drug Administration granted approval on Monday for Opzelura, which is the first topical JAK inhibitor cream for the treatment of vitiligo for people 12 and older.

This is great news for the estimated 1.9 million to 2.8 million adults in the U.S. that are living with the incurable and difficult-to-control autoimmune disorder, which is caused by antibodies that attack a person's pigment-producing cells causing patches of skin to lose color and become almost chalk white. On Black skin/darker skin tones vitiligo is more noticeable.

Treatment for those with unmet medical needs

"There's a large unmet medical need here in vitiligo," Dr. Steven Stein, Chief Medical Officer at Incyte, told ABC News. "This opens a completely new door, new avenue for them and for patients who want therapy to repigment."

"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's chief executive officer, said in a press 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."

While the discoloration caused by vitiligo doesn't typically cause medical risks, it can result in physical complications, including eye issues, hearing problems and severe sunburn. People with vitiligo also often have to face harsh reactions and judgment from others.

Another benefit of this new treatment is that it will provide vitiligo patients with something more long-term. Prior to this treatment, oral and topical steroids were a common treatment for vitiligo, however, they weren't a long-term solution for the condition.

"Since long-term use of steroids has a lot of side effects, dermatologists are always looking for steroid-sparing agents." Dr. Mansha Sethi, a board-certified dermatologist in Houston, who was not involved in the clinical trial, told ABC News.

Another treatment doctors use to treat vitiligo is phototherapy. Phototherapy works by stimulating cells to make skin pigment. The downside to this option is that it isn't always easy to implement because it can be expensive and involves going into an office equipped with phototherapy devices multiple times a week, every week.

Now with FDA approval, many people will have access to a treatment that will be covered by their insurance companies instead of having to pay hundreds of dollars out of pocket.

As for how much the medication will cost?

It's difficult to predict how much the medication will cost depending on the plan you have, however, Incyte plans to "provide copay cards that will limit the out-of-pocket cost to patients to hopefully as little as $10 or less," according to Dr. Stein. The company also has a program that some may qualify to get Opzelura for free called IncyteCARES.

How did Opzelura perform in clinical trials?

The medication, which requires a prescription and is applied as a cream to clean skin on areas affected by vitiligo twice a day was tested in two Phase 3 clinical trials, which enrolled over 600 men and women ages 12 and older.

Results of the trial showed that "adolescent and adult patients with vitiligo achieved substantial facial and total body re-pigmentation within 24 to 52 weeks of therapy."

Half of all participants had 75% improvement of their facial vitiligo after one year using the study's measurement of repigmentation.

As for side effects, those seemed to be well tolerated among patients.

"Six percent of patients will get some acne, which is usually mild. And about 6% can have an application site reaction, which could be some redness," Dr. David Rosmarin, director of the Clinical Trials Unit at Tufts, and the principal investigator for the Opzelura vitiligo clinical trials shares. "It has a very favorable safety profile, topical easy to use, and has proven to work very consistently."

Jim Lee, vice president for Incyte’s head inflammation and autoimmunity group, hopes that more doctors and patients will become aware of JAK inhibitors and more comfortable using them.

Combining the cream with other treatments, such as light therapy, to accelerate skin repigmentation may also be an option for vitiligo patients. Rosmarin says he is eager to explore these avenues in future clinical trials, and gain more knowledge on how JAK inhibitors can be used to treat other autoimmune diseases, which tend to appear in vitiligo patients.

By Cara Jones, BDO Staff Writer | Published July 20, 2022