A Closer Look at Ruxolitinib’s Pediatric AD Approval with Nicole Harter, MD

“Having a topical that has a proven track record of safety and efficacy in our adolescent population, as well as a novel mechanism of action, is something we've been really looking forward to,” Nicole Harter, MD, said in an interview with Dermatology Times.

Harter, board-certified dermatologist, fellowship program director, and division chief of pediatric dermatology at the University of Nebraska Medical Center, shared her thoughts on today’s FDA approval of topical ruxolitinib 1.5% cream (Opzelura; Incyte) for children aged 2 to 11 with atopic dermatitis (AD). She emphasized the significance of this milestone, noting that this is the first novel nonsteroidal topical agent approved for this pediatric population in nearly a decade.

Harter, who is also a member of The Society for Pediatric Dermatology, highlighted the unique value of ruxolitinib's mechanism of action as a JAK inhibitor, distinguishing it from the limited therapeutic options currently available for younger children. While previous treatments often rely on topical steroids or nonsteroidal agents with application site discomfort, ruxolitinib has shown a favorable safety and tolerability profile. Clinical studies demonstrated mostly mild adverse events, such as application site discomfort, with low discontinuation rates.

Efficacy data from clinical trials were particularly promising, as Harter noted. Patients using ruxolitinib 1.5% cream experienced statistically significant improvement in eczema severity scores and itch reduction—some as early as week 1, with continued gains through week 8. Notably, up to 75 to 90% of patients achieved substantial improvements in disease severity and pruritus. Patient-reported outcomes also reflected significant improvements in quality of life, reduced sleep disturbance, and overall well-being, which are key considerations in managing young children and their families.

Harter underscored the ongoing unmet needs in pediatric AD, stressing the limited FDA-approved therapies for children under 12. She views this approval not only as a vital new tool but also as a hopeful indicator of progress in the field of targeted therapeutics for young patients. Harter concluded with a call to action for the clinical dermatology community to educate themselves and their patients, advocate for access through insurers, and remain proactive in ensuring new therapies like ruxolitinib reach those who need them most—despite potential access challenges early in its rollout.

“We need to make sure that we are educating ourselves, our trainees, [and] our staff so that we can make sure patients are armed with the most up-to-date information and knowledge to feel that they can be treated safely and effectively and comfortably with this new agent,” Harter said.