The U.S. Food and Drug Administration (FDA) has approved ruxolitinib cream 1.5% (Opzelura, Incyte) for the short-term and non-continuous chronic treatment of mild-to-moderate atopic dermatitis (AD) in non-immunocompromised children two years of age and older whose disease is not well-controlled with topical prescription therapies, or when those therapies are not recommended.

The FDA approval of the supplemental New Drug Application (sNDA) for ruxolitinib cream was based on data from the pivotal Phase 3 TRuE-AD3 trial, which evaluated the safety and efficacy of ruxolitinib cream in children (age ≥2 to <12 years) with AD. The TRuE-AD3 study met its primary endpoint with significantly more patients treated with ruxolitinib cream achieving Investigator’s Global Assessment-treatment success (IGA-TS), a measure of treatment efficacy, than patients treated with vehicle control (non-medicated cream). 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 overall safety profile of ruxolitinib cream in the TRuE-AD3 trial was consistent with previous data, and no new safety signals were observed. No serious infections, major adverse cardiovascular events (MACE), malignancies, or thromboses were reported during the 8-week vehicle-controlled period. The most common adverse reaction was upper respiratory tract infection.

“Despite having several new non-steroidal topicals for atopic dermatitis, younger children have far fewer options. With topical ruxolitinib now approved down to age 2, this means we have real choice in this age group, and ruxolitinib has already shown itself to be highly effective in my older patients,” Peter Lio, MD, Clinical Assistant Professor of Dermatology & Pediatrics at Northwestern University Feinberg School of Medicine in Chicago, IL, tells TDD. “Importantly, having safety and efficacy data in this younger cohort helps us feel better about using it in any age group as well.”

More Experts React to New Nod

Matthew Zirwas, MD, a Dermatologist in Bexley, OH, tells TDD that, “Opzelura’s approval down to age 2 was a big win for kids with atopic dermatitis. Having a new treatment option that is nonsteroidal and that got most kids to clear or almost clear status by Week 8 in the trials is going to make a real difference, and that’s not even mentioning the rapid itch relief or long-term efficacy with as needed use.”

“As a parent of a child with atopic dermatitis, it has been challenging to see my daughter struggle growing up with her condition. There were many times her mother and I felt frustrated with the lack of treatment options that worked for our family, which was very difficult to manage as a parent,” says Adam Flores, father of Piper (age 10), who lives with AD, in a news release. “We spent years searching for the right treatment for Piper and, after joining the opzelura clinical trial, we finally saw real relief for her eczema symptoms. This approval brings hope for a new treatment to families like ours who have spent years searching for answers.”

“While every child’s journey with AD is unique, for many, the skin redness and irritation can profoundly impact their well being,” adds Korey Capozza, Founder of Global Parents for Eczema Research (GPER). “When you’re managing a condition that can affect daily life, access to safe, effective, and age-appropriate options is critical. With limited, safe treatment options currently available, especially for younger children, the addition of new therapies that control symptoms is so important to meet the needs and goals for children with AD and their families.

More About the TRuE-AD3 Trial and Ruxolitinib Cream

TRuE-AD3 is a randomized, double-blind, vehicle-controlled Phase 3 study evaluating the safety and efficacy of ruxolitinib cream compared to vehicle (non-medicated cream) in children with AD. The study enrolled over 300 patients (age ≥2 to <12 years) diagnosed with AD for at least three months and who were candidates for topical therapy.

Patients with an IGA score of 2 to 3, and with AD on 3% to 20% of their Body Surface Area (BSA; excluding scalp) were randomized 2:2:1 to receive ruxolitinib cream 0.75% administered twice daily (BID); ruxolitinib cream 1.5% BID; or vehicle (non-medicated cream) BID. Those who successfully completed an efficacy assessment at Week 8 were offered participation in the 44-week long-term safety treatment extension period with their same treatment group (ruxolitinib cream 0.75% or 1.5% BID). Patients initially randomized to vehicle cream were re-randomized (1:1) in a blinded manner to one of the active treatment groups.

The primary endpoint of TRuE-AD3 is the proportion of patients achieving an IGA-TS, defined as an IGA score of 0 (Clear) or 1 (Almost Clear) with at least a two-point improvement from baseline at Week 8. Secondary endpoints include the proportion of patients achieving at least a 75% improvement in the EASI-75 and the proportion of patients (age ≥6 to <12 years) with at least a four-point improvement in the itch numerical rating scale (NRS4 at Week 8 and time to achieve NRS4). The study also tracked the frequency, duration, and severity of adverse events associated with the use of ruxolitinib cream.

In September 2021, ruxolitinib cream, a topical Janus kinase (JAK) inhibitor was approved by the FDA for the topical short-term and non-continuous chronic treatment of mild-to-moderate AD in non-immunocompromised patients 12 years of age and older whose disease is not adequately controlled with topical prescription therapies, or when those therapies are not advisable. In July 2022, ruxolitinib cream was approved by the FDA for the treatment of nonsegmental vitiligo in adult and pediatric patients 12 years of age and older.