FDA clears Incyte's Opzelura as first vitiligo therapy

Incyte's Opzelura cream has become the first medical treatment approved to re-pigment the skin of people with vitiligo in the US, adding to its current use in atopic dermatitis.

The FDA's decision to clear the new indication for the topical JAK1/JAK2 inhibitor came after a priority review that was delayed by a request for additional data by the regulator, holding back a decision by three months.

Opzelura (ruxolitinib) is now the first alternative to steroid drugs for vitiligo, a chronic autoimmune disease that causes depigmentation of skin and affects around 1.5 million people in the US.

Steroids can be used to slow down progression of the condition, but can cause significant side effects if used too much and do nothing to restore the usual skin colour.

Approval is based on the TRuE-V1 and TRuE-V2 studies, which showed that around 30% of patients treated with ruxolitinib cream twice daily achieved 75% or more improvement from baseline in the facial vitiligo area scoring index at 24 weeks, significantly more than in a control group.

The label for the drug covers non-segmental vitiligo – the more common, autoimmune form of the condition – and continuous use of Opzelura applied twice-daily to affected areas up to 10% of total body area.

The FDA has been paying close attention to the safety of JAK inhibitors, and Opzelura was approved with black box warning that includes JAK class-wide wording for risks such as infections, heart-related events and cancer, despite being used on the skin rather than administered systemically.

It is a topical formulation of Incyte's oral drug Jakafi/Jakavi, which is FDA approved for myelofibrosis, polycythaemia vera and graft-versus-host-disease, and the new version has been billed as a major growth driver for the company.

Incyte also sells an oral JAK inhibitor, Eli Lilly-partnered Olumiant (baricitinib), for indications including rheumatoid arthritis, alopecia areata, and COVID-19.

Opzelura was approved for topic dermatitis in the US in September 2021, and according to Incyte was prescribed to more than 38,000 new patients in the first quarter of this year, driving sales of almost $13 million, which alleviated concerns that the safety issue could put a brake on uptake.

Vitiligo is key to the company's blockbuster sales projections for the product, not least because patients with vitiligo have to use more tubes of Opzelura over the course of treatment, at around ten per year versus three for atopic dermatitis.

The company is gunning for sales in atopic dermatitis alone of $1.5 billion, and while it hasn't placed a figure on the vitiligo opportunity has suggested that Opzelura could end up being used by " hundreds of thousands of patients" with the skin disorder.