FDA Approval Brings New Nonsteroidal Option for Pediatric AD

Today, the US FDA has approved ruxolitinib cream (Opzelura; Incyte) for the treatment of children aged 2 to 11 years with mild to moderate atopic dermatitis (AD). The decision, announced in a news release from the company, makes ruxolitinib the first topical Janus kinase (JAK) inhibitor approved for this pediatric population in the US.¹

This expansion builds on ruxolitinib’s prior approvals for adolescents and adults with AD and for patients 12 years and older with nonsegmental vitiligo. The new indication provides clinicians with a long-anticipated, nonsteroidal topical therapy option for younger children.

“This gives us another tool in a much-needed area,” Peter Lio, MD, a clinical assistant professor of dermatology and pediatrics at Northwestern University Feinberg School of Medicine and founding director of the Chicago Integrative Eczema Center, told Dermatology Times. “A rapid, steroid-sparing option that meaningfully reduces itch within days, helps us control flares without escalating to systemic therapy, and can help minimize the use of topical steroids.”

Addressing an Unmet Need in Pediatric AD

AD is one of the most common chronic inflammatory skin conditions in children, affecting approximately 10% of the pediatric population worldwide. Intense pruritus, recurrent flares, and sleep disturbance contribute to substantial quality-of-life impairment for both patients and families. Although topical corticosteroids remain first line, concerns about long-term use and adverse effects limit their practicality. Other steroid-sparing alternatives, such as topical calcineurin inhibitors and phosphodiesterase-4 inhibitors, can be hindered by tolerability or modest efficacy.²

“The TRuE-AD3 study showed impressive efficacy with ruxolitinib cream,” Lio told Dermatology Times. “It's always hard to know how things compare without direct head-to-head studies, but network meta-analyses that pool randomized trials suggest ruxolitinib ranks among our most effective nonsteroidal topicals. And I can say that, clinically, in my experience, it is the fastest and most effective of our nonsteroidal options.”

Evidence From TRuE-AD3

The phase 3 TRuE-AD3 study (NCT04921969), which enrolled 330 children aged 2 to 11 years with mild to moderate AD, supported the drug’s extended approval. Participants were randomly assigned to twice-daily ruxolitinib cream (0.75% or 1.5%) or vehicle for 8 weeks.³

At week 8, researchers found that significantly more children treated with ruxolitinib achieved the primary end point of Investigator’s Global Assessment treatment success (IGA-TS) compared with vehicle. Response rates were 36.6% with 0.75% cream and 56.5% with 1.5% cream vs 10.8% for vehicle (P ≤ .0001).

Secondary end points also favored ruxolitinib. More than half of patients achieved at least 75% improvement in Eczema Area and Severity Index score from baseline (51.5% with 0.75% cream and 67.2% with 1.5% cream vs 15.4% with vehicle). Improvements were observed as early as week 2 and extended to patient-reported measures, including the Patient-Oriented Eczema Measure, Infants’ Dermatitis Quality of Life Index, and Children’s Dermatology Life Quality Index.

Itch reduction, a central burden of AD, was reported early. Among patients 6 years and older, researchers stated that the median time to achieve a 4-point reduction in itch numerical rating scale scores was 11 to 13 days with ruxolitinib vs 23 days with vehicle.

"I think it is very exciting to have another nonsteroidal approved for kids with atopic dermatitis. [Ruxolitinib] has been approved in kids aged 12 years and older for vitiligo for some time now, so I do think that it is good to have multiple products approved that allow multiple options in treating atopic dermatitis," John Browning, MD, chief of dermatology at Children’s Hospital of San Antonio, told Dermatology Times’ sister publication Contemporary Pediatrics. "It's just so great to finally be able to get a 2A nonsteroidal medicine as first line, which is what I think it should be. I don't think we should have to make patients try a topical steroid first and [potentially] have a rebound issue...it is just good to finally be able to start with these."

Consistent Results Across Age Groups

Subgroup analysis confirmed consistent efficacy across younger and older children. In those aged 2 to 6 years, IGA-TS was achieved by 35.3% with 0.75% ruxolitinib and 60.6% with 1.5% vs 15.2% with vehicle. Among children aged 7 to 11 years, researchers stated that rates were 37.9% and 52.3% with ruxolitinib compared with 6.3% with vehicle.

When asked about picking a dose, Lio told Dermatology Times, “Until we get a bit more experience in the real world, I think my goal will always be to aim for the lowest effective dose that gets results.”

Safety Profile

The safety profile of ruxolitinib cream in TRuE-AD3 was consistent with findings in older populations. The most common treatment-related adverse event was mild application site pain, reported in fewer than 4% of patients. Upper respiratory tract infections and nasopharyngitis were the most frequent treatment-emergent adverse events overall, but no events indicative of systemic JAK inhibition, such as malignancies, serious infections, major adverse cardiovascular events, or thromboses, were observed.

“I explain that the boxed warning comes from a study of an oral JAK inhibitor that is different and in a different disease and population,” Lio said when asked about counseling hesitant parents. “With the cream, blood levels are low and don’t accumulate with typical use. Even in maximum-use studies and applying it to a large body surface area, plasma concentrations stay below thresholds linked to bone marrow effects. Still, I explain that it is a powerful medication and has potential adverse effects, but we consider it in the context of the overall situation and make a shared decision.”⁴

According to the company, only 2 patients discontinued treatment due to adverse events. No serious or fatal events occurred.

Clinical Significance

The FDA’s decision provides clinicians with a long-awaited corticosteroid-sparing topical therapy for children as young as 2 years. Ruxolitinib cream may help address the unmet need for effective, well-tolerated, and convenient topical options in pediatric AD.

By expanding the treatment landscape, the approval could ease disease burden, reduce reliance on corticosteroids, and improve quality of life for affected children and their families.

"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, CEO of Incyte, said in the news release. “At Incyte, we are committed to delivering innovative solutions that address every stage of a patient’s journey. This approval is another step toward addressing the real-world challenges faced by patients suffering from chronic skin conditions, including people living with atopic dermatitis.”

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 18, 2025. https://investor.incyte.com/news-releases/news-release-details/incyte-announces-additional-fda-approval-opzelurar-ruxolitinib
2. Kruse LL, Mancini AJ. Atopic dermatitis in children. Pediatr Ann. 2024;53(4):e121-e128. doi:10.3928/19382359-20240205-02
3. 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
4. Stein Gold L, Bissonnette R, Forman S, et al. A maximum-use trial of ruxolitinib cream in children aged 2-11 years with moderate to severe atopic dermatitis. Am J Clin Dermatol. 2025;26(2):275-289. doi:10.1007/s40257-024-00909-5