FDA extends review of ruxolitinib cream for pediatric atopic dermatitis

FDA extends review of ruxolitinib cream for pediatric atopic dermatitis | Image credit: Contemporary Pediatrics.

The FDA has extended its review of Incyte’s supplemental New Drug Application (sNDA) for ruxolitinib cream 0.75% (Opzelura) for the treatment of mild to moderate atopic dermatitis (AD) in children ages 2 to 11 years. The Prescription Drug User Fee Act (PDUFA) action date has been moved from June to September 19, 2025.

According to Incyte, the FDA requested additional chemistry, manufacturing, and controls (CMC) data for the 0.75% formulation of the topical Janus kinase (JAK) inhibitor. The agency will use the extended review period to evaluate the new information.

“Atopic dermatitis (AD) is a chronic immune-mediated skin condition that can be difficult to manage, particularly for the millions of children in the U.S. affected by AD,” said Steven Stein, MD, chief medical officer at Incyte. “We are confident in the potential of ruxolitinib cream to become an important non-steroidal, topical treatment option for pediatric patients with atopic dermatitis and we will continue to work closely with the FDA to ensure the Agency has all of the information needed to complete its review.”

The sNDA submission was supported by results from the phase 3 TRuE-AD3 study, which evaluated the safety and efficacy of ruxolitinib cream in children between the ages of 2 and 12 years with AD. The trial met its primary endpoint, with a significantly higher proportion of patients treated with ruxolitinib achieving Investigator’s Global Assessment-treatment success (IGA-TS) compared to those treated with vehicle cream.

The study also met a key secondary endpoint, showing that more patients in the treatment group achieved at least a 75% improvement in the Eczema Area and Severity Index (EASI75) at week 8, compared to those using the vehicle.

The safety profile of ruxolitinib cream in this younger population was consistent with prior data in older patients. No serious infections, cardiovascular events, malignancies, or thromboses were reported during the 8-week vehicle-controlled period. The most common treatment-related adverse event was mild application site pain, occurring in 2.7% of patients in the treatment group versus none in the vehicle group. No adverse events led to treatment discontinuation.

Currently, ruxolitinib is approved for the treatment of nonsegmental vitiligo and for short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis in patients aged 12 years and older. The cream is not recommended for use in combination with biologics, other JAK inhibitors, or potent immunosuppressants.

If approved for this new patient population, this would mark the first time ruxolitinib is available for pediatric patients under 12 years of age with AD.

Reference:

Incyte. Update on FDA Review of Ruxolitinib Cream (Opzelura®) for Children Ages 2-11 with Atopic Dermatitis. Incyte. June 20, 2025. Accessed June 20, 2025. https://investor.incyte.com/news-releases/news-release-details/update-fda-review-ruxolitinib-cream-opzelurar-children-ages-2-11