FDA Approves Ruxolitinib for Pediatric Patients Aged 2-11 Years as First Topical JAK Inhibitor for the Age Group

The FDA has approved the topical Janus kinase (JAK) inhibitor ruxolitinib cream (Opzelura; Incyte) for treatment of pediatric patients aged 2 to 11 years with mild-to-moderate atopic dermatitis (AD). The agency's decision, announced September 18, makes ruxolitinib the first topical JAK inhibitor approved for this pediatric age group in the US.1

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The approval expands the treatment's previous indication for adolescents aged 12 and older and adults with AD and for individuals 12 years and older with nonsegmental vitiligo. The new indication provides clinicians with a long-anticipated, nonsteroidal topical therapy option for younger children, Incyte noted in a statement.1

A "Meaningful Step Forward"

“Children currently have limited treatment options to meet their specific needs,” Peter Lio, MD, clinical assistant professor of dermatology and pediatrics at Northwestern University Feinberg School of Medicine, commented in the statement.1 “With this approval, we now have a new, nonsteroidal topical option that expands how we care for kids with this chronic disease. This is a meaningful step forward and marks a significant advancement in our ability to better support our pediatric patients.”1

Atopic dermatitis (AD) is the most common form of eczema, affecting an estimated 2–3 million U.S. children ages 2–11 and more than 21 million Americans aged 12 and older.2 Symptoms, which include itchy, inflamed, and sometimes oozing or crusted patches, can significantly disrupt quality of life and increase susceptibility to secondary infections.2 While topical steroids remain an AD treatment mainstay, chronic or uncontrolled pediatric disease often requires new therapeutic approaches, particularly for families concerned about steroid-related side effects and long-term safety. Although there are other steroid-sparing alternatives, such as topical calcineurin inhibitors and phosphodiesterase-4 inhibitors, their use can be compromised by tolerability or poor efficacy.3

TRuE-AD3 Data Support FDA Approval

TRuE-AD3 enrolled 330 children ages 2 to 11 years who had been diagnosed with mild-to-moderate AD for at least 3 months and qualified for topical therapy. Participants had disease involving 3% to 20% of their body surface area (excluding scalp) and were randomly assigned to be treated with either ruxolitinib cream (0.75% or 1.5%) or vehicle cream twice daily for 8 weeks. The primary endpoint was Investigator’s Global Assessment Treatment Success (IGA-TS) and defined as clear or almost clear skin paired with at least a 2-point improvement from baseline.4

At week 8, first author Lawrence Eichenfield, MD, and colleagues reported, results showed significantly greater response rates for children in the active drug group than in the vehicle group: 36.6% with 0.75% cream and 56.5% with 1.5% cream vs 10.8% for vehicle (P ≤.001).4

More ruxolitinib-treated children also reached the study's secondary endpoints. More than half achieved at least 75% improvement in Eczema Area and Severity Index score (EASI-75) from baseline (51.5% with 0.75% cream and 67.2% with 1.5% cream vs 15.4% with vehicle). Study authors observed improvements as early as week 2, a positive outcome that extended to patient-reported measures, including the Patient-Oriented Eczema Measure, Infants’ Dermatitis Quality of Life Index, and Children’s Dermatology Life Quality Index.4

Pruritis accounts for a substantial burden of disease for children and adults with AD, making improvement an essential test in approval of all new treatments. In TRuE-AD3, itch was reportedly quelled early. The median time to reach a 4-point reduction in itch numerical rating scale scores was 11 to 13 days for participants aged 6 years and older treated with ruxolitinib compared with 23 days for vehicle, according to the study findings.4

Importantly, the safety profile was consistent with previous adult and adolescent data, and no new safety signals were observed in the pediatric group. The most common adverse event (AE) was mild pain at the application site, observed among less than 4% of participants. The most common treatment-emergent AE were upper respiratory tract infection and nasopharyngitis. There was no evidence of serious infections, major cardiovascular events, malignancies, and blood clots during the trial,4 although the boxed warning for the JAK inhibitor class effects remains central to clinical decision-making.

Product labeling also continues to contraindicate use alongside biologics, other JAK inhibitors, or strong immunosuppressants, and comprehensive monitoring is recommended for blood counts and infection risks.1

“With this approval, we are now able to offer younger children with atopic dermatitis and their families a much-needed, steroid-free topical treatment option with the potential to significantly improve the burdensome symptoms they experience every day,” Bill Meury, Incyte chief executive officer, said in the statement.1

References

1. Incyte announces additional FDA approval of Opzelura (ruxolitinib) cream in children ages 2-11 with atopic dermatitis. News release. Incyte. September 18, 2025. Accessed September 19, 2025. https://investor.incyte.com/node/25636/pdf

2. Eczema stats. National Eczema Association. Accessed September 19, 2025. https://nationaleczema.org/eczema-facts/

3. Kruse LL, Mancini AJ. Atopic dermatitis in children. Pediatr Ann. 2024;53(4):e121-e128. doi:10.3928/19382359-20240205-02

4. Eichenfield LF, Stein Gold LF, Simpson EL, et al. Efficacy and safety of ruxolitinib cream in children aged 2 to 11 years with atopic dermatitis: results from TRuE-AD3, a phase 3, randomized double-blind study. J Am Acad Dermatol. 2025;93(3):689-698. doi:10.1016/j.jaad.2025.05.1385