FDA Approves Opzelura for Skin Condition

The FDA has approved Incyte’s Opzelura (ruxolitinib) cream for the topical treatment of vitiligo in adult and pediatric patients 12 years of age and older. Vitiligo is a chronic autoimmune disease characterized by depigmentation of skin.

Opzelura is the first FDA-approved treatment for this condition and is the only topical formulation of a Janus kinase (JAK) inhibitor approved in the United States. Over-activity of the JAK signaling pathway is believed to drive inflammation involved in vitiligo.

“Vitiligo is an immune-mediated disease that can be unpredictable, making it particularly difficult to treat,” David Rosmarin, M.D., vice chair of research and dducation, Department of Dermatology at Tufts Medical Center, said in a press release. “There have been no FDA-approved therapies available to date and the approval of Opzelura therefore marks a significant milestone.”

The approval comes after the FDA had extended the review of Opzelura to consider additional data from clinical studies. The original Prescription Drug User Fee Act (PDUFA) date was April 18, 2022.

Related: FDA Extends Review of Opzelura for Skin Condition

The approval was based on data from the pivotal phase 3 TRuE-V clinical trial program (TRuE-V1 and TRuE-V2), evaluating the safety and efficacy of Opzelura versus vehicle in more than 600 people with nonsegmental vitiligo. In the studies, treatment with Opzelura resulted in improvements in Vitiligo Area Severity Index (VASI) scores, which represent improvements in facial and total body repigmentation.

At week 24, about 30% of patients treated with Opzelura achieved 75% or more improvement in the facial VASI, the primary endpoint, compared with a non-medicated cream in 8% of patients in the TRuE-V1 study and 13% of patients in the TRuE-V2 study. At week 52, about 50% of Opzelura-treated patients achieved 75% or more improvement. Additionally, at week 24, more than 15% of patients treated with Opzelura achieved 90% or more improvement in the facial VASI. At week 52, about 30% of Opzelura-treated patients achieved this improvement.

In September 2021, Opzelura was approved by the FDA for the topical short-term treatment of mild-to-moderate atopic dermatitis in patients 12 years of age and older. This is the first topic JAK-inhibitor approved for atopic dermatitis.

Vitiligo is a disease that causes the loss of skin color. Vitiligo occurs when cells that produce melanin die or stop functioning. Vitiligo affects people of all skin types, but it may be more noticeable in people with darker skin. The exact cause is unknown, but it is believed to be an autoimmune condition.

Worldwide prevalence estimates of vitiligo vary widely, ranging from 0.004% to 2.28%. One study suggests that about 40% of patients with vitiligo may be undiagnosed.