FDA Expands Approval of Opzelura Cream to Children Ages 2 to 11 with Atopic Dermatitis

The FDA approved Opzelura (ruxolitinib) cream yesterday for the treatment of mild to moderate atopic dermatitis (AD) in children as young as two years old, marking the first topical Janus kinase (JAK) inhibitor available for pediatric patients with the chronic skin condition.

Opzelura’s manufacturer, Incyte, announced the expanded approval of the drug for short-term and non-continuous chronic use in non-immunocompromised children ages 2 through 11 whose disease is not completely controlled with other topical therapies or when those therapies are not recommended. The drug was already approved in the U.S. for use in older children and adults with atopic dermatitis as well as in patients 12 and older with nonsegmental vitiligo.

“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, chief executive officer of Incyte, said in a release.

The FDA’s decision was supported by data from the phase 3 TRuE-AD3 trial, which enrolled more than 300 children between ages 2 and 12. The study found that significantly more patients treated with Opzelura achieved Investigator’s Global Assessment-treatment success compared to those treated with a placebo cream.

Researchers also reported that a greater proportion of children using Opzelura reached at least a 75% improvement in the Eczema Area and Severity Index at week 8.

In addition, safety outcomes in the trial were consistent with earlier studies in older patients, with no new safety concerns observed. No cases of serious infection, malignancy, cardiovascular events or blood clots were reported. The most common reaction was upper respiratory tract infection.

The trial also included a long-term extension phase, allowing researchers to monitor patients for up to 44 weeks to better understand the durability of response and ongoing safety of Opzelura in a pediatric population. These results provide further confidence in the treatment’s risk-benefit profile.

“Navigating a complex condition like atopic dermatitis can be very challenging for children, who currently have limited treatment options to meet their specific needs,” Peter Lio, M.D., clinical assistant professor of dermatology and pediatrics at Northwestern University Feinberg School of Medicine, said in the release.

AD, the most common form of eczema, is a chronic disease that causes red, itchy skin prone to irritation, lesions and secondary infection. In the U.S., the condition affects an estimated 2 to 3 million children between ages 2 and 11 and more than 21 million adolescents and adults.

For many families, the new approval represents both relief and hope.

“As a parent of a child with atopic dermatitis, it has been challenging to see my daughter struggle growing up with her condition,” Adam Flores, father of 10-year-old Piper, said in the release, adding that this approval “brings hope for a new treatment to families like ours who have spent years searching for answers.”

An Incyte spokesperson told Managed Healthcare Executive that the dose and cost of Opzelura are the same for children as for adults. They added that the company is working with insurers and pharmacy benefit managers (PBMs) to expand access for eligible patients and continue to offer its Opzelura On Trac support program, which helps families navigate coverage, refills and affordability.

With the new approval, Opzelura becomes the first steroid-free topical JAK inhibitor available to children as young as two, highlighting an important milestone in the treatment of AD.