Daily Upadacitinib Pill May Restore Skin Colour in Some People With Vitiligo

A daily tablet has helped some people with vitiligo recover skin colour. Vitiligo is a condition that produces white patches on the skin.

This development may provide a simpler treatment, especially for people whose vitiligo covers extensive or difficult-to-reach parts of the body.

Rather than being applied directly to separate patches as creams are, the tablet acts across the whole body.

Vitiligo occurs when the immune system wrongly attacks the cells responsible for skin colour. Although it cannot be passed from person to person, its visible patches can affect everyday life and mental health.

People with vitiligo may face stigma, anxiety, and social exclusion. For that reason, restoring skin colour involves more than appearance.

Existing options include creams and light therapy. While these can help skin regain colour, they commonly involve repeated treatment over many months. Creams may also be challenging to use when vitiligo affects large areas.

A UV photograph of a hand affected by vitiligo. (Chip Bobbert/CC BY-SA 4.0)

Upadacitinib trials for vitiligo

A new study published in The Lancet examined a medicine called upadacitinib in 614 adults and teenagers aged 12 or over.

The two trials took place at 138 institutions across 18 countries. Every participant had the most common type of vitiligo, with patches on both the face and body.

Participants were randomly allocated to receive either 15 milligrams of upadacitinib or a placebo daily for 48 weeks. Neither the participants nor the researchers evaluating their skin knew who had received the actual medicine.

In the first trial, 25 percent of patients given upadacitinib recovered at least 75 percent of the colour lost from their face. The equivalent result was achieved by only 6 percent of participants receiving a placebo.

Results from the second trial were nearly the same: 23 percent of patients on the drug met the target, versus 7 percent in the placebo group.

Colour also returned in other affected body areas. Across the two trials, 19 percent and 21 percent of patients receiving upadacitinib regained at least half of the colour lost from these areas. In each placebo group, only 6 percent had the same degree of improvement.

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A proportion of patients had more pronounced improvements. Across both trials, 14 percent of treated patients recovered at least 90 percent of the colour lost from their face, compared with 2 to 3 percent of placebo recipients.

At the end of the 48-week trials, skin was continuing to regain colour, indicating that a longer course of treatment might deliver additional benefits.

How the daily pill works

Upadacitinib dampens a part of the immune response that drives vitiligo. This could lessen the attack on healthy skin cells, allowing affected areas the chance to regain colour.

The medicine is part of a class known as JAK inhibitors. Other medicines in this class have also been investigated for conditions where an overactive immune response damages the skin.

As the pill works throughout the body, it could be particularly useful for people with widespread vitiligo or patches in locations where applying creams is difficult. It may also be more convenient than making repeated journeys to a clinic for light therapy.

Upadacitinib has now been approved in the European Union for eligible adults and teenagers aged 12 or over with this common form of vitiligo. Its approval status differs in other places.

Uncertainties and safety concerns

Important questions remain. Around three-quarters of patients taking the tablet did not meet the main goal for facial improvement, while approximately four in five did not recover at least half of the colour lost over their body.

The most frequently reported issues were upper respiratory infections, acne, headaches, and inflammation of the nose and throat. Seven patients who took the drug developed shingles. Serious health problems were reported in 4 percent of treated patients in the first trial and 2 percent in the second.

The 48-week comparison was too short to exclude uncommon or long-term risks. When used for other conditions, upadacitinib already carries warnings concerning serious infections, cancer, heart problems, and blood clots.

Participants will continue to be monitored for a further 112 weeks, which could show how much more improvement is achievable.

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It also remains unknown whether restored colour persists once treatment ends.

The trials measured the pill against a placebo rather than existing creams or light therapy. Most participants were White, with people who have darker skin underrepresented.

AbbVie, which manufactures upadacitinib, funded the study. The company contributed to the trial design, analysed the data, and took part in preparing the paper.

The results offer early evidence that a daily tablet can restore a meaningful degree of lost skin colour for some people. Longer studies will show how durable these improvements are and assist doctors in identifying who is most likely to benefit safely.

The study was published in The Lancet.

This article was fact-checked by Fiona MacDonald and edited by Peter Dockrill. While we take pride in our process, we are only human. If you spot an error, please let us know.