20 July 2022 - - The US Food and Drug Administration 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, US-based Incyte (NASDAQ: INCY) said.

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.

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 pyrexia.

The labeling for Opzelura includes a Boxed Warning for serious infections, mortality, malignancy, major adverse cardiovascular events and thrombosis.

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 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 1.5m people are diagnosed with vitiligo2. The overall prevalence of the condition is estimated to be approximately 2-3 m3, with the majority of patients (approximately 85%) suffering from nonsegmental vitiligo.

Vitiligo can occur at any age, although many patients with vitiligo will experience initial symptoms before the age of 305.

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 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.

Incyte is committed to supporting patients and removing barriers to access medicines.

Eligible patients in the United States who are prescribed Opzelura have access to IncyteCARES (Connecting to Access, Reimbursement, Education and Support), a program offering patient support, including financial assistance and ongoing education and resources to eligible patients.

The TRuE-V clinical trial program includes two Phase 3 studies, TRuE-V1 (NCT04052425) and TRuE-V2 (NCT04057573), evaluating the safety and efficacy of ruxolitinib cream in patients with vitiligo.

Each study enrolled approximately 300 patients (age ≥12 years) who have been diagnosed with nonsegmental vitiligo.

Opzelura, a novel cream formulation of Incyte's selective JAK1/JAK2 inhibitor ruxolitinib, is the first and only topical JAK inhibitor approved for use in the United States, indicated for the topical treatment of nonsegmental vitiligo in adult and pediatric patients 12 years of age and older and the topical short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis 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.

Use of Opzelura in combination with therapeutic biologics, other JAK inhibitors, or potent immunosuppressants, such as azathioprine or cyclosporine, is not recommended.

In October 2021, Incyte announced the validation of the European Marketing Authorization Application for ruxolitinib cream as a potential treatment for adolescents and adults (age >12 years) with nonsegmental vitiligo with facial involvement.

Incyte has worldwide rights for the development and commercialization of ruxolitinib cream, marketed in the United States as Opzelura.

Incyte is a Wilmington, Delaware-based, global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics.