FDA Approves Ruxolitinib Cream as First Repigmentation Therapy for Vitiligo

Ruxolitinib (Opzelura) cream is the first and only FDA-approved treatment for repigmentation in patients with vitiligo.

FDA has approved ruxolitinib (Opzelura) cream 1.5% for the treatment of nonsegmental vitiligo in adult and pediatric patients 12 years of age and older. Incyte, the maker of ruxolitinib, announced the approval in a statement.

This is the first therapy to receive FDA approval for repigmentation in patients with vitiligo. Approval is based on data of the pivotal phase 3 TRuE-V clinical trial program (TRuE-V1 [NCT04052425] and TRuE-V2 [NCT04057573]), which evaluated the safety and efficacy of ruxolitinib cream vs vehicle (nonmedicated cream) in more than 600 people with nonsegmental vitiligo and depigmented areas covering less than or equal to 10% total body surface area (BSA).

Findings of the primary analysis at week 24 showed that a significantly greater proportion of patients randomized to 1.5% ruxolitinib cream twice daily (BID) achieved the primary and secondary end points vs vehicle BID, including a greater than or equal to 75% improvement from baseline in the facial Vitiligo Area Scoring Index (F-VASI75) and greater than or equal to 50% improvement from baseline in total body VASI (T-VASI50).

Further data from a 28-week treatment extension of the initial 24-week TRuE-V1 and TRuE-V2 studies presented as a late-breaking session at the 2022 American Academy of Dermatology (AAD) Annual Meeting showed ruxolitinib cream monotherapy achieved the primary and secondary end points in a significantly greater proportion of patients across 52 weeks vs 24 weeks and baseline, with overall safety consistent with previous study data.

These efficacy data included greater proportions of patients at week 52 vs week 24 achieving F-VASI75 (50% vs 29.9%), greater than or equal to 50% improvement from baseline in the F-VASI (F-VASI50) (75% vs 51%), greater than or equal to 90% improvement from baseline in the F-VASI (F-VASI90) (30% vs 15%), T-VASI50, Vitiligo Noticeability Scale (VNS) response, and improvement on percentage change from baseline in facial BSA.

Patients who were switched from vehicle BID to ruxolitinib cream bid for the 28-week treatment extension were also shown to exhibit efficacy data consistent with week 24 data in patients who were randomized to the intervention group from baseline. Incyte's statement noted that satisfactory patient response with ruxolitinib cream may require treatment for more than 24 weeks.

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

“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.”

The most common adverse reactions associated with ruxolitinib cream included application site acne, application site pruritus, nasopharyngitis, headache, urinary tract infection, application site erythema, and pyrexia. The labeling for the drug also has a Boxed Warning for serious infections, mortality, malignancy, major adverse cardiovascular events, and thrombosis.

This is the second indication for ruxolitinib cream which was approved by the FDA in September 2021 for the topical short-term and noncontinuous chronic treatment of mild to moderate atopic dermatitis in nonimmunocompromised patients 12 years of age and older whose disease is not adequately controlled with topical prescription therapies, or when those therapies are not advisable.

Ruxolitinib cream serves as the only topical formulation of a Janus kinase inhibitor approved in the United States.