Therapy Areas: Autoimmune
US FDA Accepts for Priority Review sNDA for Ruxolitinib Cream as a Treatment for Patients with Vitiligo
17 December 2021 - - The US Food and Drug Administration has accepted for Priority Review the supplemental New Drug Application for ruxolitinib cream 1.5% (Opzelura) a topical JAK inhibitor, as a potential treatment for adolescents and adults (age ≥12 years) with vitiligo, US-based Incyte (NASDAQ: INCY) said.

The FDA grants Priority Review to medicines that may offer a major advance in treatment where none currently exists. The Prescription Drug User Fee Act (PDUFA) target action date is April 18, 2022.

The sNDA is supported by data from the Phase 3 TRuE-V clinical trial program evaluating the safety and efficacy of ruxolitinib cream in more than 600 people with non-segmental vitiligo, age 12 and older.

Results from the Phase 3 program were recently presented at the 30th European Academy of Dermatology and Venereology congress during a late-breaking research session.

The data showed that at Week 24, 29.9% of patients applying ruxolitinib cream achieved ≥75% improvement from baseline in the facial Vitiligo Area Scoring Index (F-VASI75), the primary endpoint.

In September 2021, Opzelura (ruxolitinib) cream 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.

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

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.

It affects approximately 1.5m+ people in the US 1 and there are no US Food and Drug Administration -approved drug therapies for repigmentation in vitiligo.

It can occur at any age, although many patients with vitiligo will experience initial symptoms before the age of 20.

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.

The studies each enrolled approximately 300 patients (age ≥12 years) who have been diagnosed with non-segmental vitiligo and have depigmented areas including at least 0.5% of the body surface area on the face, ≥0.5 facial Vitiligo Area Scoring Index [F-VASI] score, at least 3% BSA on nonfacial areas, ≥3 total body Vitiligo Area Scoring Index [T-VASI] score and total BSA involvement (facial and nonfacial) of up to 10%.

Participants were randomized into two arms: 1.5% ruxolitinib cream twice daily and vehicle control for the 24-week double-blind period.

Patients who successfully completed baseline and Week 24 assessments, including those that received vehicle control during the double-blind phase, were offered treatment extension with 1.5% ruxolitinib cream BID for an additional 28 weeks.

The primary endpoint of both studies in the TRuE-V program is the proportion of patients achieving F-VASI75, defined as at least a 75% improvement from baseline in the F-VASI score at Week 24.

Key secondary endpoints include: the proportion of patients achieving F-VASI50 (at least 50% improvement from baseline in the F-VASI), F-VASI90 (at least 90% improvement from baseline in the F-VASI) and T-VASI50 (at least 50% improvement from baseline in the T-VASI) at Week 24, the proportion of patients achieving a Vitiligo Noticeability Scale score of 4 (a lot less noticeable) or 5 (no longer noticeable) at Week 24, and the percentage change from baseline in facial BSA (F-BSA) at Week 24.

The studies also track the frequency, duration and severity of adverse events associated with the use of ruxolitinib cream.

Ruxolitinib cream (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 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.

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

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

On October 28, 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 non-segmental vitiligo with facial involvement.
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