FDA Approves 1.5% Ruxolitinib Cream for Pediatric Atopic Dermatitis

FDA officials approved 1.5% ruxolitinib cream (Opzelura; Incyte) for the treatment of atopic dermatitis (AD) in children aged 2 to less than 12 years.¹ The approval marks a new option for patients with this often-irritating condition.

This marks the first topical Janus kinase (JAK) inhibitor approved in the US for pediatric AD and is the third US approval for ruxolitinib cream 1.5%. It is also indicated to treat mild to moderate AD in nonimmunocompromised patients 2 years and older and nonsegmental vitiligo in patients 12 years and older.¹

“With this approval, we are now able to offer younger children with atopic dermatitis and their families and much-needed, steroid-free topical treatment option with the potential to significantly improve the burdensome symptoms they experience every day,” said Bill Meury, CEO of Incyte, in a news release.¹

About Atopic Dermatitis

Pediatric AD, commonly known as eczema, is a chronic inflammatory skin condition that typically presents in infancy or early childhood. It is characterized by intensely itchy, red, and scaly patches of skin, often appearing on the face, scalp, extensor surfaces in infants, and flexural areas (elbows and knees) in older children.²

The underlying cause of AD is complex and involves a combination of genetic predisposition, environmental triggers, and immune system dysfunction. Children with AD often have a compromised skin barrier, which makes them more susceptible to irritants, allergens, and infections. In turn, this leads to a cycle of itching and scratching, which further exacerbates AD. Pediatric AD can significantly affect the child’s quality of life, sleep, and emotional well-being, and cause stress for their families and caregivers.^3,4

“While every child’s journey with AD is unique, for many, the skin redness and irritation can profoundly impact their well-being,” said Korey Capozza, founder of Global Parents for Eczema Research, in the news release. “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.”¹

Management and treatment of pediatric AD focuses on a multi-pronged approach designed to control symptoms, prevent flares, and improve skin barrier function. Cornerstone therapies include consistent moisturizing with emollients to hydrate the skin and repair the barrier, and topical corticosteroids to reduce inflammation during flares. For more severe cases or those unresponsive to conventional treatments, calcineurin inhibitors or crisaborole may be considered.⁵

Ruxolitinib cream, a topical JAK inhibitor, offers a nonsteroidal option, now available for children aged 2 and older with mild to moderate AD whose disease is not adequately controlled with other topical prescription therapies or when those therapies are not advisable. Ruxolitinib targets the signaling pathways of key cytokines that contribute to the inflammation and itch associated with AD.¹

“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, MD, a clinical assistant professor of dermatology and pediatrics at Northwestern University Feinberg School of Medicine, said in the news release. “With this approval, we now have a new, nonsteroidal topical option that expands how we care for kids with this chronic disease. This is a meaningful step forward and marks a significant advancement in our ability to better support our pediatric patients.”¹

Ruxolitinib Clinical Trials

The TRuE-AD3 (NCT04921969) trial is the primary trial supporting the use of ruxolitinib cream in pediatric patients with AD. It is a randomized, double-blind, vehicle-controlled phase 3 study evaluating the safety and efficacy of ruxolitinib cream compared to vehicle in children with AD. The study enrolled over 300 patients aged 2 to less than 12 years diagnosed with AD for at least 3 months and who were candidates for topical therapy.^6,7

According to results announced in 2023, the trial met its primary end point with significantly more patients treated with ruxolitinib cream 0.75% and 1.5% achieving Investigator’s Global Assessment Treatment Success (IGA-TS) than patients treated with vehicle control. IGA-TS is defined as an IGA score of 0 (clear) or 1 (almost clear) with at least a 2-point improvement from baseline at week 8.⁶

In the trial, 56.5% of patients treated with ruxolitinib cream 1.5% and 36.6% treated with ruxolitinib cream 0.75% achieved IGA-TS at week 8 (P <= .0001 for both) compared to 10.8% of those treated with vehicle. Furthermore, more than half of patients treated with ruxolitinib cream (67.2% of those with 1.5% cream; 51.5% of those with 0.75% cream) achieved Eczema Area and Severity Index 75 (EASI75) compared to those treated with vehicle at week 8 (P < .0001 for both).⁷

Among patients 6 to less than 12 years old, time to Numerical Rating Scale ≥4-point (NRS4) improvement was achieved by 43.4% and 37.5% of patients in the ruxolitinib cream 1.5% and ruxolitinib cream 0.75% arms, respectively, at week 8 compared to 29.7% of those treated with vehicle. The median time to NRS4 was 13 and 11 days in the 1.5% and 0.75% arms, respectively, compared to 23 days for vehicle (hazard ratio, 1.74/1.77; P < .05 for both).⁷

Results from the maximum-use trial (MUsT) in children age 2 to less than 12 years with at least 35% of their body surface area affected by AD showed that ruxolitinib cream was well tolerated, with efficacy results consistent with data from an adolescent and adult maximum-use study and a pilot pharmacokinetics and safety pediatric study. In the MUsT trial, 20.7% of patients treated with ruxolitinib cream 1.5% reported treatment emergent adverse events (TEAEs) through week 8; none were serious or led to treatment interruption or discontinuation, and no TEAEs suggestive of systemic JAK inhibition were reported.⁷

“AD is a chronic immune-mediated disease that impacts about 13% of children in the US, yet there remains a need for new treatment options to help this age group manage this difficult to treat skin condition,” Lawrence Eichenfield, MD, chief of pediatric and adolescent dermatology at Rady Children’s Hospital San Diego, said in an October 2023 news release. “As a clinician, I have been extremely pleased with the results achieved by many of my adolescent and adult patients with AD prescribed ruxolitinib cream, and I am excited about the potential to have a safe, well tolerated, and effective nonsteroidal topical treatment option available to my pediatric patients in the future.”⁷

The Pharmacist’s Role

Pharmacists play a crucial role in supporting the effective management of pediatric AD, particularly with ruxolitinib cream is part of the treatment plan. Although direct monitoring of topical adherence can be challenging, pharmacists can employ practical strategies to gauge compliance.

In an interview with Pharmacy Times, Matthew Zirwas, MD, said one of the easiest ways to monitor compliance with topicals is by asking the patient where they are applying it and how often. For instance, if a patient says they are applying it to their face once per day, that should take approximately 1 g per day, and they should be out of the medication in approximately 1 month.⁸

“It’s not that patients are lying whenever they say, ‘Oh, I use it every day,’” Zirwas said. “But in reality, they didn’t use it; they just think they used it. So, getting at it that way…that can be one of the ways to help gauge compliance.”⁸

Pharmacists are also instrumental in helping patients and their families overcome the significant financial and access barriers associated with high-cost medications like ruxolitinib cream. Zirwas noted that the list price can be in the range of $2000 per tube, so even patients with a 20% copay will often find that unaffordable. To address this, pharmacists can proactively identify and collaborate with local pharmacies or mail-order pharmacies to guide patients through the prior authorization process. Additionally, pharmacists can leverage information provided in e-prescriptions, such as the location of the AD on the body and past prescriptions that have been ineffective, as these details are crucial for securing prior authorization approvals.⁸

Furthermore, pharmacists can guide patients toward available patient support programs, including those offered by Incyte, the manufacturer of Opzelura, which provides a copay program to help eligible patients with their out-of-pocket costs for Opzelura.⁹ By connecting patients with these resources and assisting with the complex paperwork involved in prior authorizations and patient assistance applications, pharmacists significantly improve the likelihood of patients accessing this treatment.

“The biggest thing that surprised me about Opzelura for atopic dermatitis is the difference it makes in patients’ lives,” Zirwas said. “It’s even bigger than I would have expected…it’s incredibly effective, but it’s the first drug for AD that has given patients a sense of control over their disease.”⁸

REFERENCES

1. Incyte Announces Additional FDA Approval for Opzelura (Ruxolitinib) Cream in Children Ages 2-11 with Atopic Dermatitis. News release. Incyte Therapeutics. September 18, 2025. Accessed September 19, 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 June 17, 2025. https://www.stanfordchildrens.org/en/topic/default?id=atopic-dermatitis-in-children-90-P01675

3. Johnson JL. The complex science behind itch in atopic dermatitis. National Eczema Association. Updated January 3, 2023. Accessed June 17, 2025. https://nationaleczema.org/blog/science-of-itch/

4. Burden of Atopic Dermatitis. American Academy of Pediatrics. Updated June 11, 2021. Accessed June 17, 2025. https://www.aap.org/en/patient-care/atopic-dermatitis/burden-of-atopic-dermatitis/#:~:text=The%20distress%20of%20persistent%20pruritus,stress%20and%20tension%20between%20parents

5. Frazier W, Bhardwaj N. Atopic dermatitis: diagnosis and treatment. Am Fam Physician. 2020;101(10):590-598.

6. Incyte Announces Positive Topline Results From Phase 3 Trial Evaluating Ruxolitinib Cream (Opzelura) In Children With Atopic Dermatitis. News release. Incyte. July 11, 2023. Accessed June 17, 2025. https://investor.incyte.com/news-releases/news-release-details/incyte-announces-positive-topline-results-phase-3-trial

7. Incyte Announces New Data for Ruxolitinib Cream (Opzelura) In Children With Atopic Dermatitis. News release. Incyte. October 13, 2023. Accessed June 17, 2025. https://investor.incyte.com/news-releases/news-release-details/incyte-announces-new-data-ruxolitinib-cream-opzelurar-children

8. Understand Effective Strategies for Monitoring Topical Dermatological Agents. Pharmacy Times. March 18, 2025. Accessed June 17, 2025. https://www.pharmacytimes.com/view/understand-effective-strategies-for-monitoring-topical-dermatological-agents

9. Opzelura Copay Savings Card. Accessed June 17, 2025. https://www.opzelurahcp.com/atopic-dermatitis/savings#:~:text=Eligible*%20patients%20with%20commercial%20insurance,%240%20per%20tube%20for%20OPZELURA