FDA approves Ruxolitinib (Opzelura) for Vitiligo Therapy: A breakthrough in the field of dermatology
The U.S. Food and Drug Administration (FDA) approved Opzelura™ (ruxolitinib) cream 1.5% on July 18, 2022, for the topical treatment of nonsegmental vitiligo in adults and pediatric patients aged 12 years and older. Developed by Incyte, Opzelura is the first and only FDA-approved therapy specifically indicated for repigmentation in vitiligo and represents the only topical Janus kinase (JAK) inhibitor approved in the U.S. for this condition. Vitiligo is a chronic autoimmune disease characterized by the loss of melanocytes, the pigment-producing cells in the skin, resulting in white patches. Nonsegmental vitiligo, which affects approximately 85% of vitiligo patients, is the most common form and can be particularly challenging to treat.
Ruxolitinib cream’s approval was supported by results from the Phase 3 TRuE-V1 and TRuE-V2 clinical trials, which included over 600 patients aged 12 and older. The studies demonstrated that treatment with ruxolitinib cream led to significant improvements in facial and total body repigmentation, measured by the Facial Vitiligo Area Scoring Index (F-VASI). At Week 24, about 30% of patients treated with ruxolitinib cream achieved at least 75% improvement in facial repigmentation (F-VASI75), compared to just 8% and 13% of those treated with a non-medicated vehicle cream. Continued treatment showed even more promising results, with approximately 50% of patients reaching F-VASI75 by Week 52. Additionally, the proportion of patients achieving F-VASI90 (≥90% improvement) increased from 15% at Week 24 to about 30% at Week 52.
Ruxolitinib cream is approved for continuous use twice daily to affected areas covering up to 10% of body surface area. It may require more than 24 weeks of treatment for a satisfactory response. The most common side effects reported were mild and included application site acne, itching, erythema, headache, nasopharyngitis, urinary tract infections, and fever. However, the product includes a boxed warning for serious risks, such as infections, malignancy, major cardiovascular events, thrombosis, and mortality.
The approval of ruxolitinib cream marks a major advancement for patients with vitiligo who previously had no FDA-approved treatment options for repigmentation. Dr. David Rosmarin, Vice Chair of Research and Education at Tufts Medical Center’s Department of Dermatology, described this milestone as a significant breakthrough, noting the difficulty in treating an unpredictable disease like vitiligo. Ruxolitinib cream was previously approved in September 2021 for the short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis in non-immunocompromised patients aged 12 years and older.
Patients in the United States prescribed ruxolitinib cream may also have access to support programs offering financial assistance, reimbursement guidance, and educational resources to ensure easier access to treatment. The approval represents a crucial step forward in dermatology, bringing new hope to individuals affected by vitiligo.
Reference: https://investor.incyte.com/news-releases/news-release-details/incyte-announces-us-fda-approval-opzeluratm-ruxolitinib-cream-0
