FDA Approves Ruxolitinib Cream for Pediatric Patients with Atopic Dermatitis

The US Food and Drug Administration (FDA) has approved ruxolitinib cream (OPZELURA) for the treatment of children aged 2 - 11 years with moderate-to-severe atopic dermatitis, according to a recent press release from Incyte Corporation.1

The September 18, 2025, announcement indicates ruxolitinib cream for the short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis in non-immunocompromised children ≥ 2 years of age whose disease is not well controlled with topical prescription therapies, or when those therapies are not recommended. The decision follows recent positive clinical trial findings from the phase 3 TRuE-AD3 trial.1

"I think it is very exciting to have another non-steroidal approved for kids with atopic dermatitis," John Browning, MD, Chief of Dermatology at Children’s Hospital of San Antonio and Organon Health Partner, said in a statement to HCPLive's sister publication Contemporary Pediatrics. "[Ruxolitinib] has been approved in kids aged 12 years and older for vitiligo for some time now, so I do think that it is good to have multiple products approved that allow multiple options in treating atopic dermatitis."

Ruxolitinib was designed to block the JAK1 and JAK2 enzymes, both of which are known to maintain a key role in cytokine and growth factor signaling pathways. These pathways drive inflammation and immune responses in those with atopic dermatitis, so the drug’s targeting of the pathways may help to address inflammation and immune-related damage to patients’ skin resulting from atopic dermatitis.

The FDA’s decision to approve ruxolitinib cream follows the positive clinical findings of the phase 3 TRuE-AD3 study and others, presented as a poster during the Revolutionizing Atopic Dermatitis (RAD) Conference in Nashville, TN.2 The TRuE-AD3 study had involved children aged 2-11 years with mild to moderate disease, using a randomized, double-blind, vehicle-controlled study design.

The investigative team randomly assigned the subjects 2:2:1 to be given ruxolitinib cream twice daily at concentrations of 0.75%, 1.5%, or vehicle for a total of 8 weeks of continuous treatment. This was later followed by 44 weeks of as-needed treatment.

The drug’s implementation led to maintenance of disease control and similar safety findings over 52 weeks in children aged 2-6 years and 7-11 years with the skin disease. Patients were shown by investigators to have spent almost half of the long-term treatment period off the drug due to the clearance of lesions.

In the study's safety analysis, both ruxolitinib cream concentrations were noted by investigators as having been well tolerated and to have few application site reactions.2 In 56.00% of subjects, treatment-emergent adverse events (TEAEs) took place in the aged 2-6 years cohort receiving 0.75% cream. TEAEs were also noted among 63.4% of those receiving 1.5% cream.

Among the 7-11 year-old patient subgroup, it had been noted that 45.2% reported TEAEs with 0.75% cream and 55.6% reported such events with 1.5% cream. In their assessment of the most common TEAEs, respiratory tract infection were observed in 16.0% of those aged 2-6 years and 11.9% of those aged 7-11 years on 0.75% cream. It was also noted among 13.4% of those aged 2-6 years and 16.7% aged 7-11 years on 1.5% cream.

There were very few application site reactions observed, no serious TEAEs seen over the 52-week study's course, and no new safety signals.

Ruxolitinib cream 1.5% has already been given approval by the FDA for the short-term, non-continuous treatment of pediatric patients aged 12 years and older with mild to moderate atopic dermatitis.3 The current approval allows for more widespread use of the therapeutic option for patients with this disease as well as others.

"It's just so great to finally be able to get a 2A non-steroidal medicine as first line, which is what I think it should be," Browning said. "I don't think we should have to make patients try a topical steroid first and [potentially] have a rebound issue... it is just good to finally be able to start with these."

References

1. Incytye. Incyte Announces Additional FDA Approval of Opzelura® (Ruxolitinib) Cream in Children Ages 2-11 with Atopic Dermatitis. September 18, 2025. Accessed September 18, 2025. https://investor.incyte.com/news-releases/news-release-details/incyte-announces-additional-fda-approval-opzelurar-ruxolitinib
2. Incytye. Update on FDA Review of Ruxolitinib Cream (Opzelura®) for Children Ages 2-11 with Atopic Dermatitis. June 20, 2025. https://www.businesswire.com/news/home/20250620553806/en/Update-on-FDA-Review-of-Ruxolitinib-Cream-Opzelura-for-Children-Ages-2-11-with-Atopic-Dermatitis.
3. Sturm D, Eichenfield E, et al. 52-Week Disease Control and Safety With As-Needed Application of Ruxolitinib Cream in Children Aged 2 to 11 Years With Moderate and/or More Extensive Atopic Dermatitis: Subgroup Analysis From the TRuE-AD3 Study. Poster presented at: 2025 Revolutionizing Atopic Dermatitis Conference; June 6-7, 2025; Nashville, TN.
4. Incyte Announces U.S. FDA Approval of Opzelura™ (ruxolitinib) Cream, a Topical JAK Inhibitor, for the Treatment of Atopic Dermatitis (AD). Incyte. September 21, 2021. https://investor.incyte.com/news-releases/news-release-details/incyte-announces-us-fda-approval-opzeluratm-ruxolitinib-cream. Accessed June 20, 2025.