FDA Extends Review Period of Ruxolitinib Cream for Children with Atopic Dermatitis

The US Food and Drug Administration (FDA) has extended its period of review for ruxolitinib cream (OPZELURA) as a treatment for children ages 2 - 11 years with mild to moderate atopic dermatitis, despite the previous target action date having been set for June 2025.1

The June 20 announcement by Incyte indicates that the new Prescription Drug User Fee Act (PDUFA) target action date has been pushed to September 19, 2025. Ruxolitinib cream’s supplemental New Drug Application (sNDA) had been accepted for review and its PDUFA originally slated for June 2025, following positive findings in the phase 3 TRuE-AD3 trial demonstrating the drug’s efficacy in pediatric patients with atopic dermatitis.2

“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,” Incyte’s Chief Medical Officer, Steven Stein, MD, said in a statement.1 “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 treatment is a novel cream formulation of Incyte’s selective JAK1/JAK2 inhibitor ruxolitinib, and it is known to work through the inhibition of JAK1 and JAK2 enzymes. The medication is already the first FDA-approved drug for repigmentation in those aged 12 years and older with non-segmental vitiligo. Its review extension follows the FDA’s request for additional data related to manufacturing, chemistry, and controls for the 0.75% formulation of the cream.

Inctye’s announcement suggests that the agency is allowing extra time to evaluate this supplemental information.1 The sNDA is supported by data from the phase 3 TRuE-AD3 trial, which looked into the safety and effectiveness of ruxolitinib cream in pediatric patient with the skin disease.2 The trial was successful in achieving its primary endpoint. Investigators found that a significantly higher proportion of children treated with ruxolitinib cream attaining Investigator’s Global Assessment-treatment success (IGA-TS) versus those using the vehicle cream.

Additionally, the team on the TRuE-AD3 study found that a key secondary endpoint was me among patients: a significantly greater percentage of those evaluated were successful in achieving at least a 75% improvement from the point of baseline in their Eczema Area and Severity Index (EASI75) scores at the 8-week mark.

In their assessment of ruxolitinib cream’s safety outcomes in the TRuE-AD3 trial, the investigators noted that their findings aligned with previously reported ones. Specifically, they identified no unexpected safety issues in their analysis of the drug. Within the 8-week period in which the analysis was vehicle-controlled, no reports of major adverse cardiovascular events (MACE), serious infections, malignancies, or thrombotic events were identified.

The trial investigators further concluded that the most common treatment-related adverse event in the group treated with ruxolitinib cream was application site pain. This, they highlighted, occurred in 2.7% of those assessed, compared to 0% in the vehicle arm of the study. Such instances were also noted as mild and did not result in the discontinuation of ruxolitinib cream.

References

1. Update on FDA Review of Ruxolitinib Cream (Opzelura®) for Children Ages 2-11 with Atopic Dermatitis. Incyte. June 20, 2025. https://www.businesswire.com/news/home/20250620553806/en/Update-on-FDA-Review-of-Ruxolitinib-Cream-Opzelura-for-Children-Ages-2-11-with-Atopic-Dermatitis.

2. Sturm D, Eichenfield E, et al. 52-Week Disease Control and Safety With As-Needed Application of Ruxolitinib Cream in Children Aged 2 to 11 Years With Moderate and/or More Extensive Atopic Dermatitis: Subgroup Analysis From the TRuE-AD3 Study. Poster presented at: 2025 Revolutionizing Atopic Dermatitis Conference; June 6-7, 2025; Nashville, TN.