FDA approves Opzelura for repigmentation in nonsegmental vitiligo

The U.S. Food and Drug Administration (FDA) has approved Opzelura (ruxolitinib) cream 1.5% for the topical treatment of nonsegmental vitiligo in adult and pediatric patients 12 years of age and older. Opzelura is the first and only FDA-approved treatment for repigmentation in patients with vitiligo, and the only topical formulation of a Janus kinase (JAK) inhibitor approved in the United States. Vitiligo is a chronic autoimmune disease characterized by depigmentation of skin.

"With the approval of Opzelura in nonsegmental vitiligo, Incyte has once again delivered a treatment to patients with high unmet medical need who previously had no approved therapies," said Hervé Hoppenot, Chief Executive Officer, Incyte. "We are proud of Incyte's scientists and development teams that have made this milestone possible, and we're pleased that eligible vitiligo patients now have a choice to address repigmentation."

In patients with non-segmental vitiligo, Opzelura is approved for continuous topical use twice daily to affected areas of up to 10% body surface area. Satisfactory patient response may require treatment with Opzelura for more than 24 weeks.

The FDA 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, age 12 and older.

In the studies, treatment with Opzelura resulted in significant improvements in VASI scores, which represent improvements in facial and total body repigmentation at Week 24 (primary analysis) compared to vehicle (non-medicated cream) and in an open-label extension at Week 52.

Results at Week 24, which were consistent across both studies, showed that approximately 30% of patients treated with Opzelura achieved ≥75% improvement from baseline in the facial Vitiligo Area Scoring Index (F-VASI75), the primary endpoint, compared to approximately 8% and 13% of patients treated with vehicle in TRuE-V1 and TRuE-V2, respectively. At Week 52, approximately 50% of Opzelura-treated patients achieved F-VASI75.

Additionally, at Week 24, more than 15% of patients treated with Opzelura achieved ≥90% improvement from baseline in F-VASI (F-VASI90), compared to approximately 2% of patients treated with vehicle. At Week 52, the percentage of Opzelura-treated patients who achieved F-VASI90 doubled to approximately 30%.

In the vehicle controlled period of the Phase 3 studies, the most common adverse reactions (incidence ≥ 1%) are application site acne, application site pruritus, nasopharyngitis, headache, urinary tract infection, application site erythema, and pyrexia1. The labeling for Opzelura includes a Boxed Warning for serious infections, mortality, malignancy, major adverse cardiovascular events and thrombosis. See additional Important Safety Information below.

Week 52 data from the Phase 3 TRuE-V studies were featured in an oral presentation at the late-breaking abstract session at the American Academy of Dermatology (AAD) Annual 2022 Meeting.

Vitiligo is a chronic autoimmune disease characterized by depigmentation of skin that results from the loss of pigment-producing cells known as melanocytes. Over-activity of the JAK signaling pathway is believed to drive inflammation involved in the pathogenesis and progression of vitiligo. In the United States, more than 1.5 million people are diagnosed with vitiligo2.

The overall prevalence of the condition is estimated to be approximately 2-3 million3, with the majority of patients (approximately 85%) suffering from nonsegmental vitiligo4. Vitiligo can occur at any age, although many patients with vitiligo will experience initial symptoms before the age of 305.

"Vitiligo is an immune-mediated disease that can be unpredictable, making it particularly difficult to treat," said David Rosmarin, M.D., Vice Chair of Research and Education, Department of Dermatology at Tufts Medical Center. "There have been no FDA-approved therapies available to date and the approval of Opzelura therefore marks a significant milestone. I welcome a medical treatment that helps my patients with nonsegmental vitiligo who are interested in potentially reversing the depigmentation caused by their disease."

In September 2021, Opzelura was approved by the FDA for the topical short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis (AD) in non-immunocompromised patients 12 years of age and older whose disease is not adequately controlled with topical prescription therapies, or when those therapies are not advisable.