FDA Approves Topical Ruxolitinib for AD in Children Aged 2 and Up

The US Food and Drug Administration (FDA) has approved ruxolitinib cream 1.5% (Opzelura) for the short-term and non-continuous chronic treatment of mild-to-moderate atopic dermatitis (AD) in non-immunocompromised children ages 2 and older whose disease is inadequately controlled with topical prescription therapies or when those therapies are not advisable, according to Incyte.

The company said in a press release that the approval was based on results from the Phase 3 TRuE-AD3 trial, which evaluated the safety and efficacy of the topical JAK inhibitor in children aged ≥2 to <12 years with AD. The study met its primary endpoint, with a significantly greater proportion of patients achieving Investigator’s Global Assessment-treatment success (IGA-TS) compared to vehicle (non-medicated cream). A key secondary endpoint—achievement of EASI 75 (≥75% improvement in the Eczema Area and Severity Index) at Week 8—was also met.

No new safety signals were reported in the trial. The most common adverse event was upper respiratory tract infection. No serious infections, malignancies, major adverse cardiovascular events (MACE), or thrombotic events were observed during the 8-week vehicle-controlled treatment period.

“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,” Incyte CEO Bill Meury said in the press release. “This approval is another step toward addressing the real-world challenges faced by patients suffering from chronic skin conditions.”

Peter Lio, MD, Clinical Assistant Professor of Dermatology and Pediatrics at Northwestern University Feinberg School of Medicine, called the approval “a meaningful step forward,” noting that younger children have long had limited options for safe and effective treatment.

Ruxolitinib was first approved in 2021 for the treatment of mild-to-moderate AD in patients aged 12 and older, and in 2022 for the treatment of nonsegmental vitiligo in patients 12 and older.

“Atopic dermatitis can affect every aspect of a child’s life,” said Korey Capozza, Founder of Global Parents for Eczema Research. “Access to safe, effective, and age-appropriate options is critical.”