Opzelura From Incyte Corp.

The FDA approved 1.5% ruxolitinib cream (Opzelura; Incyte) for the treatment of atopic dermatitis (AD) in children aged 2 to less than 12 years, offering a new treatment option for pediatric patients with this inflammatory skin condition.¹

Nearly 2 million to 3 million patients aged 2 to 11 years are impacted by pediatric AD, a chronic skin condition that often affects infants and children with symptoms emerging between 3 and 6 months of age. Symptoms in infants often affect the face, neck, scalp, elbows, and knees. In children, symptoms often appear inside the elbows, on the backs of the knees, the sides of the neck, around the mouth, and on the wrists, ankles, and hands. Although these symptoms can vary, they often present as dry, scaly skin; severe itching, redness, and swelling; thickened skin; pale skin on the face; small, raised bumps that leak fluid; rough skin; darkened skin of eyelids or around the eyes; and hives.²

Children who have family members with AD are at greater risk, as researchers have identified a genetic link. Children’s underdeveloped immune systems and external factors, such as colder weather, hot water when bathing, soap, dry skin, and hot temperatures, put children at greater risk. Children with AD have a weakened skin barrier that increases their vulnerability to irritants, allergens, and infections, creating an itching and scratching cycle that worsens symptoms. The condition can greatly affect a child’s quality of life, sleep, and emotional health, also causing stress for families and caregivers.²

Pharmacology and Pharmacokinetics

Ruxolitinib cream 1.5% is the first Janus kinase (JAK) inhibitor approved in the US for pediatric AD. The topical treatment is also approved for repigmentation in patients 12 years and older with nonsegmental vitiligo.¹

Dosage and Administration

Ruxolitinib cream 1.5% is indicated as a short-term and noncontinuous topical chronic treatment for mild to moderate eczema in nonimmunocompromised adults and children 2 years and older whose disease is not well controlled with other topical prescription therapies or when therapies are not recommended.¹ A thin layer should be applied to affected skin areas, and patients or caregivers should wash their hands before and after applying the cream. The treated area should not be covered with a bandage or other occlusive dressing unless on the instructions of a physician.

Clinical Trials

The FDA approval in children with AD is based on data from the randomized, double-blind, vehicle-controlled phase 3 TRuE-AD3 trial (NCT04921969) that evaluated the safety and efficacy of the cream compared with a nonmedicated cream in children with AD. A total of 300 patients aged 2 to 12 years diagnosed with AD for at least 3 months were included in the study. Patients with mild to moderate AD, defined by an investigator’s global assessment (IGA) score of 2 to 3 and 3% to 20% body surface area involvement were randomly assigned 2:2:1 to receive ruxolitinib cream 0.75% twice daily, ruxolitinib cream 1.5% twice daily, or a nonmedicated vehicle cream twice daily. Those who completed the 8-week efficacy phase could continue in a 44-week long-term safety extension using the same treatment. After that, patients who initially received the vehicle cream were again randomly assigned to be in one of the active treatment groups.^1,3

The results demonstrated that the study met its primary end point, with significantly more patients using ruxolitinib cream achieving IGA treatment success compared with individuals using a nonmedicated vehicle cream. It also met a key secondary end point, showing more patients achieved at least a 75% improvement in their eczema area and severity index by week 8.¹

The most common adverse event was upper respiratory tract infection; no serious adverse cardiovascular events, malignancies, or thromboses were reported during the 8-week vehicle-controlled period.¹

Contraindications, Warnings, and Precautions

Ruxolitinib cream 1.5% carries several warnings related to its effects on the immune system. As a JAK inhibitor, it can increase the risk of serious infections, including tuberculous, bacterial, fungal, or viral infections, and should not be used in individuals with active infections. Ruxolitinib can cause low blood cell counts and increased cholesterol levels, so regular monitoring by a health care provider is recommended during treatment.¹

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 October 6, 2025. https://investor.incyte.com/news-releases/news-release-details/incyte-announces-additional-fda-approval-opzelurar-ruxolitinib

2. Atopic dermatitis in children. Stanford Medicine Children’s Health. Accessed October 6, 2025. https://www.stanfordchildrens.org/en/topic/default?id=atopic-dermatitis-in-children-90-P01675

3. Soong W, Zaenglein A, Tollefson M, et al. Efficacy and safety of ruxolitinib cream among children with atopic dermatitis aged 2 to 6 years and 7 to <12 years: results from a phase 3 double-blind vehicle-controlled study. J Allerg Clin Immunol. 2024;153(suppl 2):AB1. doi:10.1016/j.jaci.2023.11.027