U.K. Regulators May Approve Controversial Vitiligo Cream

Officials from the United Kingdom could possibly approve ruxolitinib, a cream restoring the skin's pigment, which is associated with rare but severe side effects.

Sold under the brand Opzelura, the drug restores the skin's natural color. The U.K. National Institute for Health and Care Excellence's (NICE) website lists the medicine's status as "awaiting development," with an expected release date on February 28, 2024. If approved, the drug could be used in patients 12 years and older.

Vitiligo is a chronic autoimmune disease where melanocytes — skin cells that make pigment — are attacked and destroyed, causing patches of skin to lose pigment or color.

Non-segmental vitiligo is the most common form of the condition, characterized by pale patches on both sides of the body that may affect a large area. Segmental vitiligo only affects one side of the body and is most common in young children.

Worrying side effects

The FDA first authorized ruxolitinib — an active ingredient of Opzelura — in September 2021 to treat mild eczema and later expanded the approval for non-segmental vitiligo in individuals 12 and older.

The FDA's decision was based on two Phase 3 clinical trials, TRuE-V1 and TRuE-V2, that included over 600 participants with non-segmental vitiligo affecting less than 10% of their bodies. In the trials, about one-third of participants using the 1.5% ruxolitinib cream twice daily saw significant improvements in the repigmentation of vitiligo lesions.

Ruxolitinib is a selective Janus kinase (JAK) inhibitor that works by blocking enzymes called JAK1 and JAK2, both of which contribute to inflammation.

The most common side effects of Opzelura were mild, including acne, itching, and redness on the application site. The drug may make patients vulnerable to common colds, headaches, urinary tract infections, and fevers. However, the FDA issued a black box warning on the Opzelura, which alerts about serious side effects, such as injury or death.

According to the label, in the case of Opzelura, serious infections, non-melanoma skin cancers, and thrombosis have occurred in vitiligo patients treated with the drug. The medication is also associated with blood disorders, such as anemia (a lack of enough healthy red blood cells), thrombocytopenia (too low platelet count in the blood), and neutropenia (a low number of white blood cells).

Despite the possible adverse reactions, Opzelura may also reach pharmacy shelves of European Union countries. In February, the European Medicines Agency issued a positive opinion recommending the approval of ruxolitinib cream for the treatment of non-segmental vitiligo with facial involvement of individuals from 12 years of age. The opinion is now being reviewed by the European Commission.