The US FDA has approved ruxolitinib (Opzelura) cream 1.5% for the short-term and noncontinuous chronic treatment of mild-to-moderate atopic dermatitis (AD) in nonimmunocompromised children aged 2 years or older whose disease is not well controlled with topical prescription therapies, or when those therapies are not recommended.

In September 2021, the FDA approved topical ruxolitinib for the same indications for children and adults aged 12 years or older.

This is the first topical JAK inhibitor approved in the US for pediatric AD, the manufacturer, Incyte, said in a September 18 press release announcing the expanded approval. In the US, AD affects an estimated 2-3 million people aged 2-11 years and more than 21 million people aged 12 years or older, according to the company.

Approval was based on data from the pivotal phase 3 TRuE-AD3 trial, a randomized, double-blind, vehicle-controlled trial that evaluated the safety and efficacy of ruxolitinib cream in children aged 2-11 years with AD.

“The TRuE-AD3 study met its primary endpoint with significantly more patients treated with Opzelura achieving Investigator’s Global Assessment treatment success, a measure of treatment efficacy, than patients treated with vehicle control,” according to the release. “In addition, a secondary endpoint of patients demonstrating at least a 75% improvement in the Eczema Area and Severity Index (EASI-75) at week 8 was also achieved,” the company reports.

Safety in the study was consistent with previous data, with no new safety signals. During the 8-week vehicle control period, no serious infections, major adverse cardiovascular events (MACE), malignancies, or thromboses were reported. Upper respiratory tract infections were the most common adverse event, according to the release.

Labeling for topical ruxolitinib and oral JAK inhibitors includes a boxed warning regarding serious infections, mortality, malignancy, MACE, and thrombosis. “Some people have had serious infections while taking JAK inhibitors by mouth, including tuberculosis (TB), and infections caused by bacteria, fungi, or viruses that can spread throughout the body. Some people have been hospitalized or died from these infections,” the company states in its press release, adding that healthcare providers should watch closely for signs and symptoms of TB during treatment.

Ruxolitinib cream is also approved in the US to treat nonsegmental vitiligo in patients aged 12 years or older.

Marcia Frellick is an independent, Chicago-based healthcare journalist and a regular contributor to Medscape Medical News.