FDA approves first at-home therapy for skin repigmentation in vitiligo patients

Patients with vitiligo will now have access to the first ever at-home therapy for skin repigmentation approved by the Food and Drug Administration.

The FDA announced the approval of Incyte’s ruxolitinib cream on Monday based on data collected from two duplicate Phase 3 clinical trials that found 30% of patients using the cream regained 75% or more skin repigmentation on the face and roughly 20% of patients regained at least 50% or more repigmentation on their body after 24 weeks.

Jim Lee, vice president for Incyte’s head inflammation and autoimmunity group, said before the approval of ruxolitinib, whose brand name is Opzelura, vitiligo patients were limited to light therapies administered in a doctor’s office. Now, they can administer a cream at home.

“It’s very exciting and quite an honor to be able to bring the first treatment option for these patients,” Lee said.

Vitiligo affects millions of people; an estimated 0.5% to 2% of the worldwide population has the autoimmune disorder.

David Rosmarin, a dermatologist at Tufts Medical Center and lead investigator of the clinical trial of the therapy, said roughly half of the patients who develop vitiligo see symptoms by the age of 20. To understand how adolescents would tolerate the drug, one-tenth of the study participants enrolled were between the ages of 12 and 17.

Berardo Rivas first discovered white spots on his knees and chest around the age of 10. He said he’s tried vitamins, topical creams, and ultraviolet light therapy, some of which helped, while others had “zero effect.”

Depigmentation on his face, arms, and hands were the most frustrating for him. During his time in the Navy, Rivas worked in a high-stress environment as an air traffic controller, and said the depigmentation spread more quickly. When faced with the decision of whether to renew his contract with the military, Rivas said he chose his health.

By 2020, Rivas said he had given up finding a vitiligo treatment, until he learned of Incyte’s clinical trial from a friend who saw the recruitment ad on social media. He enrolled and began using the ruxolitinib cream, which he said absorbs into the skin within minutes.

After the yearlong, double-blind clinical study, and another year using the ruxolitinib cream during the open-label extension for trial participants, Rivas said he has less than 5% of depigmentation on his face and about 20% on the rest of his body.

“In my face, I just have two small dots,” Rivas said. “From going from looking like a raccoon to having nothing on my face is a miracle.”

The drug works by quelling patients’ overactive immune systems; it binds to Janus-associated kinase (JAK) receptors on the surface of the immune cell intent on destroying skin cells, or melanocytes. Once the immune cells are inhibited, new melanocytes are able to grow into the area and, slowly, the pigment returns to patients’ skin.

Due to the slow repigmentation process, Rosmarin said some patients need more time to see the benefits of the ruxolitinib cream. From six months to one year of use, the percent of patients who experienced at least 75% of repigmentation on their face increased from 30% to nearly 50%.

It’s unclear what will happen to patients if they stop using the cream or use it less frequently than the prescribed twice-daily application, a regime that Rivas said required discipline. “It’s not one of those things that you put on today and tomorrow you’re going to get results,” he said. “It’s nothing but patience and consistency … that you will get your future results.”

But Rivas is not daunted by the prospect of using the cream forever, if need be. “I’m going to continue using it until I have no more vitiligo, or at least … contain it to where it doesn’t spread as rapidly any more,” he said.

It may be possible to combine the cream with other treatments, such as light therapy, to accelerate skin repigmentation. Rosmarin said he is eager to explore these avenues in future clinical trials, and to understand how JAK inhibitors can be used to treat other autoimmune diseases, which tend to appear in vitiligo patients.

Another group of patients — the roughly 20% of vitiligo patients with depigmentation on more than 10% of their total body area — were not included in Incyte’s clinical trial. The FDA and Incyte agreed those patients might be better suited with a systemic treatment. Although, Lee said, the entire face accounts for only 3-4% of total skin area, meaning the clinical trial included patients with full face depigmentation.

The FDA recently approved another JAK inhibitor drug developed by Incyte, baricitinib, which is a pill taken to treat hair loss caused by alopecia areata. Although the mechanism the pill, now owned by Eli Lilly, is based on is similar to that of the ruxolitinib cream, Lee said the pill is more effective at treating cells found deeper in the skin, such as hair cells.

Lee hopes as more drugs using JAK inhibitors to treat autoimmune disorders go on the market, doctors and patients will be more aware of them and more comfortable using them. Lee and Rosmarin agree widespread adoption depends on making the cream accessible to patients.

“Vitiligo is not a cosmetic disease,” Rosmarin said. “It can have a big impact on [patients], so we want to make sure that patients have access to the medicine who want to use it.”