FDA Approves Ruxolitinib for Nonsegmental Vitiligo


Ruxolitinib (Opzelura; Incyte) cream 1.5% is the first and only treatment approved by the FDA for repigmentation in patients with vitiligo.

The FDA has approved ruxolitinib (Opzelura; Incyte) cream 1.5% for the topical treatment of nonsegmental vitiligo in adult and pediatric patients 12 years of age and older. The action makes the Janus kinase (JAK) inhibitor the first and only treatment approved by the FDA for repigmentation in patients with vitiligo.

“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, in a press release. “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.”

Ruxolitinib inhibits JAK1 and JAK2, which mediate the signaling of several cytokines and growth factors important for hematopoiesis and immune function. Downstream, JAK receptor stimulation involves modulating signals that recruit signal transducers and activators of transcription (STATs) after phosphorylation at the JAK receptors.

Phosphorylated STATs move to the nucleus to modulate gene transcription of cytokines. However, specific JAK enzymes’ importance in treatment effectiveness is currently unknown.

The FDA previously approved ruxolitinib in September 2021for 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.

In patients with non-segmental vitiligo, ruxolitinib was approved for continuous topical use twice daily to affected areas of up to 10% body surface area. Incyte noted that satisfactory patient response may require ruxolitinib treatment for longer than 24 weeks.

The latest approval was based on data from the phase 3 TRuE-V clinical trial (TRuE-V1 and TRuE-V2), which analyzed the safety and efficacy of ruxolitinib compared with vehicle (non-medicated cream) in more than 600 patients aged 12 years and older with nonsegmental vitiligo. The trial investigators found that treatment with ruxolitinib produced significant improvements in VASI scores, which signify improved facial and total body repigmentation at week 24 (primary analysis) versus non-medicated cream and in an open-label extension at week 52.

Both studies found that at week 24, approximately 30% of patients administered ruxolitinib achieved ≥75% improvement from baseline in the facial Vitiligo Area Scoring Index (F-VASI75), which was the primary endpoint, versus approximately 8% and 13% of patients treated with non-medicated cream in TRuE-V1 and TRuE-V2, respectively. At week 52, approximately 50% of patients administered ruxolitinib achieved F-VASI75.

Further, more than 15% of patients administered ruxolitinib achieved ≥90% improvement from baseline in F-VASI (F-VASI90) at week 24, versus approximately 2% of patients administered non-medicated cream. At week 52, the percentage of patients who achieved F-VASI90 during treatment with ruxolitinib doubled to approximately 30%.

In the vehicle-controlled period of both studies, the most common adverse events (incidence ≥ 1%) were application site acne, application site pruritus, nasopharyngitis, headache, urinary tract infection, application site erythema, and pyrexia. Ruxolitinib’s labeling includes a Boxed Warning for serious infections, mortality, malignancy, major adverse cardiovascular events, and thrombosis. Ruxolitinib’s labeling recommends against combining it with therapeutic biologics, other JAK inhibitors, or potent immunosuppressants, such as azathioprine or cyclosporine.

“Vitiligo is an immune-mediated disease that can be unpredictable, making it particularly difficult to treat,” said David Rosmarin, MD, vice chair of Research and Education, Department of Dermatology at Tufts Medical Center, in a press release. “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.”


Incyte Announces U.S. FDA Approval Of Opzelura™ (Ruxolitinib) Cream For The Treatment Of Vitiligo. Incyte. News release. July 18, 2022. https://investor.incyte.com/news-releases/news-release-details/incyte-announces-us-fda-approval-opzeluratm-ruxolitinib-cream-0

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