FDA Approves First Topical Treatment for Vitiligo

The Food and Drug Administration (FDA) on July 19 approved Opzelura (ruxolitinib), the first and only topical treatment for vitiligo in patients 12 years of age and older.

Dermatologists said the approved medication is a huge advancement for patients with vitiligo and it can lead to repigmentation of the skin.

“The dermatology community is so excited that we finally have something effective, FDA-approved, and readily available and easy to use for our patients with vitiligo,” Amy Spizuoco, DO, FAOCD, a board-certified dermatologist in New York, told Verywell. “It’s groundbreaking and going to be life-changing for people who are suffering from a chronic autoimmune condition.” 

Opzelura is owned and manufactured by Incyte Dermatology. Jim Lee, MD, PhD, vice president at Incyte’s head inflammation and autoimmunity group, told Verywell in an email that the company is working with insurance providers in the United States to ensure access for eligible patients.

Why This New Treatment Matters 

Based on data from two Phase 3 clinical trials, 30% of the participants who used Opzelura regained at least 75% skin repigmentation on their face.

Prince Adotama, MD, a dermatologist at NYU Langone Health, told Verywell that approval of Opzelura offers a different and less invasive option to help treat vitiligo, beyond phototherapy and topical steroids.

“Phototherapy requires you to leave the home two or three times a week on a regular basis and steroids can have harmful side effects, so this new treatment gives patients a lot more control,” Adotama said.

In addition, he said this new treatment may improve the quality of life for kids and adults who are affected by the condition.

“Vitiligo affects a lot of people and even patients of color who tend to have darker skin complexion. These white patches can be very stigmatizing and isolating and can make patients feel uncomfortable,” Adotama said.

Berardo Rivas, who’s had vitiligo since he was 10, was enrolled in the Phase 3 trial for Opzelura.

“I had vitiligo around my eyes, eyebrows, lips, elbows, and knees. I started seeing results at three months and then it started progressing,” Rivas told Verywell. “At my year mark, I have about 50% of facial repigmentation back on my skin. Now, at almost two years, I have less than 5% of vitiligo on my skin.”

After the yearlong trial, he has continued the treatment for over two years now.

“I’ve tried numerous treatments from UV lighting, other topical creams, and even vitamins, but Opzelura is the only treatment that has worked for me and it has made my life much better,” Rivas said. “Assuming it worked for me brings hope that something out there can work for others who have vitiligo as well.”

How Does Opzelura Work?

Opzelura is a cream for topical use only, according to the FDA. Patients who are prescribed to use the medication by their healthcare provider or dermatologist should apply a thin layer of Opzelura twice a day to affected areas of up to 10% of the body’s surface area.

The cream is considered to be a topical Janus kinase (JAK) inhibitor, which is an anti-inflammatory treatment, according to Spizuoco.

“JAK inhibitors tend to be safer in the long term than steroids," Spizuoco said. "We don’t have to worry about some of the overuse side effects that we can see from topical steroids such as thinning of the skin, enlarged blood vessels, and stretch marks."

Lee added that Opzelura works by blocking immune cells from destroying melanocytes, the pigment-producing cell in the skin. “By blocking this autoimmune reaction, the melanocytes can re-pigment the skin.”

However, it can take as long as 24 weeks of consistent use of the cream before patients can see results.

"I applied the cream twice a day, eight hours apart for the last two years, so it’s a pain,” Rivas said. “But it’s also not going to be a treatment where you put it on today and you’re going to see results tomorrow.”

At this time, it’s unclear whether patients who are using Opzelura need to continue treatment beyond 24 weeks or longer, especially if repigmentation is occurring. 

“We don’t have the data yet to show what that looks like, but if patients reach the limit and then go back on the medication, it would be perfectly acceptable for them,” Spizuoco said. 

Known Side Effects 

The most common adverse reactions or side effects of taking Opzelura include pain or swelling in the nose or throat, diarrhea, bronchitis, ear infection, increase in a type of white blood cell (eosinophil) count, hives, inflamed hair pores, swelling of the tonsils, and runny nose.

The labeling for the cream also includes a boxed warning for serious bacterial, viral, and fungal infections; lymphomas and other cancers; cardiovascular events including heart attack and stroke; thrombosis; and increased all-cause mortality.

These are not all of the possible side effects of using Opzelura. Patients should discuss with their healthcare provider about the risks and benefits if they are considering this treatment.