Incyte Receives Positive CHMP Opinion for Opzelura® Cream in Adults with Moderate Atopic Dermatitis
Incyte has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the approval of Opzelura® (ruxolitinib) cream for the treatment of moderate atopic dermatitis (AD) in adult patients whose disease cannot be adequately managed with topical corticosteroids (TCSs) or topical calcineurin inhibitors (TCIs), or for whom those therapies are not appropriate.
The recommendation marks an important regulatory milestone for the company and brings Opzelura one step closer to becoming the first non-steroidal topical Janus kinase (JAK) inhibitor approved in the European Union for adults with moderate atopic dermatitis who have limited treatment options.
If the European Commission (EC) grants final marketing authorization, Opzelura would expand therapeutic choices for patients living with chronic eczema by offering a targeted topical treatment designed to control inflammation and relieve symptoms without the use of corticosteroids.
The positive opinion is supported by data from several Phase 3 clinical trials demonstrating meaningful improvements in disease severity, itch, skin clearance, and quality of life while maintaining a favorable safety profile.
A Significant Need for New Topical Therapies
Atopic dermatitis is the most common form of eczema and is a chronic inflammatory skin disease characterized by persistent itching, redness, dryness, and recurrent skin lesions.
Although topical corticosteroids remain the standard first-line treatment for many patients, long-term use can be associated with limitations, including concerns regarding skin thinning and other corticosteroid-related adverse effects.
Topical calcineurin inhibitors offer another treatment option but may not provide adequate disease control for every patient and are not suitable in all clinical situations.
Consequently, many adults continue to experience persistent symptoms despite currently available topical therapies, affecting daily activities, sleep, work productivity, emotional well-being, and overall quality of life.
Incyte believes Opzelura has the potential to fill this important treatment gap by providing a non-steroidal alternative for patients who require additional therapeutic options before progressing to systemic therapies.
Company Highlights Importance of Positive Recommendation
Lee Heeson, Executive Vice President and Head of Incyte International, described the CHMP’s recommendation as an important step toward improving treatment options for adults living with moderate atopic dermatitis across Europe.
He noted that the chronic nature of the disease often places a considerable burden on patients, affecting both physical health and daily functioning.
According to Heeson, the positive opinion reflects meaningful progress toward introducing the first topical JAK inhibitor for adults in Europe whose disease has not responded adequately to conventional topical treatments.
He added that, if approved by the European Commission, Opzelura could help address an unmet need for patients who have exhausted or cannot tolerate traditional topical corticosteroids or calcineurin inhibitors.
Recommendation Supported by Robust Phase 3 Clinical Program
The CHMP based its positive opinion primarily on results from the pivotal Phase 3 TRuE-AD4 clinical study.
The recommendation was further supported by findings from the earlier Phase 3 TRuE-AD1 and TRuE-AD2 studies, which collectively evaluated the efficacy and safety of ruxolitinib cream in adults with atopic dermatitis.
Together, these trials provide a comprehensive body of evidence demonstrating the therapy’s ability to reduce disease severity, improve skin appearance, relieve itching, and enhance patient quality of life.
The studies enrolled adults with moderate atopic dermatitis who had experienced inadequate responses, intolerance, or contraindications to both topical corticosteroids and topical calcineurin inhibitors.
Rapid Improvement in Disease Symptoms
One of the notable findings from the TRuE-AD4 study was the rapid onset of clinical benefit.
Patients treated with ruxolitinib cream experienced significant improvements in both the visible signs of atopic dermatitis and the intense itching associated with the condition.
Importantly, reductions in itch were observed as early as Day 2 following treatment initiation, highlighting the therapy’s potential to provide early symptom relief.
The ability to rapidly reduce itching is particularly important because persistent itch represents one of the most burdensome symptoms experienced by patients with atopic dermatitis and often contributes to sleep disruption, skin damage from scratching, and reduced quality of life.
Primary Endpoints Successfully Achieved
The Phase 3 study successfully met both of its primary efficacy endpoints at Week 8.
A significantly greater proportion of patients treated with ruxolitinib cream achieved at least a 75% improvement in the Eczema Area and Severity Index (EASI75) compared with those receiving vehicle cream.
The trial also demonstrated statistically significant improvements in Investigator’s Global Assessment Treatment Success (IGA-TS).
Treatment success on this measure required patients to achieve either completely clear skin or almost clear skin, together with at least a two-point improvement from baseline.
These results indicate that ruxolitinib cream was able to substantially improve overall disease severity in adults with moderate atopic dermatitis.
Sustained Clinical Benefits Through 24 Weeks
Clinical improvements observed during the initial treatment period were maintained over longer-term follow-up.
At Week 24, approximately 84.3% of patients receiving ruxolitinib cream on an as-needed basis had achieved EASI75.
Similarly, 70.6% of patients achieved Investigator’s Global Assessment Treatment Success during continued treatment.
The studies also demonstrated substantial reductions in the amount of skin affected by eczema.
Mean body surface area involvement decreased from 15.1% at baseline to 2.5% by Week 8, with this improvement maintained through Week 24.
These findings suggest that many patients experienced durable disease control with continued intermittent use of the cream.
Meaningful Relief from Itch
In addition to improving visible skin lesions, ruxolitinib cream provided sustained relief from itching.
Using the Itch Numeric Rating Scale (NRS4), researchers measured the proportion of patients achieving at least a four-point improvement in itch severity.
Approximately 74.3% of patients reached this threshold by Week 8.
The benefit remained substantial throughout extended treatment, with 64.7% of patients continuing to experience clinically meaningful itch improvement at Week 24.
Because chronic itching is often considered one of the most disruptive symptoms of atopic dermatitis, these findings may represent an important benefit for patients seeking long-term symptom control.
Improved Quality of Life
The Phase 3 program also demonstrated that clinical improvements translated into meaningful gains in patients’ daily lives.
Researchers assessed health-related quality of life using the Dermatology Life Quality Index (DLQI).
Patients treated with ruxolitinib cream experienced a reduction in mean DLQI scores from 19.3 at baseline to 4.3 at Week 8.
By comparison, patients receiving vehicle cream improved from 19.1 to 10.7 during the same period.
These improvements suggest that better disease control was accompanied by reduced disruption to work, social activities, emotional well-being, and everyday functioning.
Favorable Safety Profile
The safety findings from the clinical development program were consistent with previous experience using ruxolitinib cream.
Throughout the 24-week treatment period, investigators reported no serious infections, major adverse cardiovascular events, malignancies, or thrombotic events associated with treatment.
The most frequently observed treatment-emergent adverse events were upper respiratory tract infections, reported in 10.6% of patients, and nasopharyngitis, reported in 6.3%.
Overall, the therapy demonstrated good tolerability while maintaining clinically meaningful efficacy across multiple outcome measures.
Dermatology Expert Welcomes Potential New Option
Dr. Andreas Wollenberg, Professor of Dermatology and Allergy at University Hospital Augsburg in Germany, commented that many adults with moderate atopic dermatitis continue to experience inadequate disease control despite available topical therapies.
He noted that persistent symptoms can significantly affect patients’ quality of life and often lead physicians to consider systemic treatment when topical options fail.
According to Dr. Wollenberg, the clinical evidence supporting the CHMP recommendation indicates that ruxolitinib cream could provide an effective new topical alternative for appropriate patients in Europe.
He also suggested that earlier access to an effective non-steroidal therapy may help delay or even prevent the need for systemic medications in some individuals.
Understanding the Burden of Atopic Dermatitis
Atopic dermatitis affects up to 4% of adults worldwide, with prevalence estimates ranging between 4.4% and 7.1% among adults in Europe.
The condition is characterized by chronic inflammation, severe itching, recurrent skin lesions, redness, and dryness.
Symptoms frequently fluctuate over time, with periods of worsening disease followed by temporary improvement.
Beyond its physical manifestations, atopic dermatitis can significantly impair sleep quality, mental health, workplace productivity, and overall quality of life.
The need for additional effective and well-tolerated topical therapies remains an important priority within dermatology.
European Commission Decision Expected
Following the CHMP’s positive recommendation, the application will now be reviewed by the European Commission, which has the authority to grant centralized marketing authorization throughout the European Union.
If approved, this would become the second European indication for Opzelura.
The medicine has already received European Commission approval for the treatment of non-segmental vitiligo with facial involvement in adults and adolescents aged 12 years and older.
Adding moderate atopic dermatitis would further expand the role of Opzelura as a targeted topical treatment for immune-mediated skin disorders.
The CHMP’s positive opinion represents a major step toward expanding treatment options for adults with moderate atopic dermatitis whose disease remains inadequately controlled by conventional topical therapies. Supported by a comprehensive Phase 3 clinical program, Opzelura demonstrated rapid itch relief, significant improvements in skin clearance, durable disease control, enhanced quality of life, and a favorable safety profile.
If the European Commission grants final approval, Opzelura could become the first non-steroidal topical JAK inhibitor authorized in the European Union for this patient population, offering physicians and patients a new targeted approach for managing moderate atopic dermatitis while potentially reducing reliance on corticosteroids and delaying the need for systemic therapies.
About TruE-AD4
TRuE-AD4 (NCT06238817) is a randomized, double-blind, vehicle-controlled Phase 3b study designed to evaluate the efficacy and safety of Opzelura ® (ruxolitinib) cream in adults with moderate atopic dermatitis (AD). The study enrolled 241 patients (≥18 years old) who met the specific inclusion criteria, including an IGA (Investigator’s Global Assessment) score of 3 and an EASI (Eczema Area and Severity Index) score greater than 7 at both screening and Day 1, and who have AD on 10% to 20% of their Body Surface Area (BSA; excluding scalp). Patients also had to have a documented history of inadequate response, intolerance, or contraindication to topical corticosteroids (TCSs) and topical calcineurin inhibitors (TCIs) within the 12 months prior to the screening visit.
Patients were randomized 2:1 to receive Opzelura applied twice daily (BID) or vehicle (non-medicated cream) BID. At Week 8, patients who achieved EASI50 continued double-blind, BID treatment, applied as needed for an additional 16 weeks. Patients for whom EASI50 was not achieved at two consecutive visits ≥1 week apart were eligible to enter the escape arm at Week 8 or later, in which open-label Opzelura cream was applied as needed.
The co-primary endpoints of TRuE-AD4 were the proportion of patients achieving IGA Treatment Success (IGA-TS), defined as an IGA score of 0 (clear) or 1 (almost clear) with at least a two-point improvement from baseline, and EASI75, defined as ≥75% improvement in EASI score, at Week 8.
Secondary endpoints included the proportion of patients with a ≥4-point improvement in NRS4 (≥4-point improvement in Itch Numeric Rating Scale) score at various time points. Other efficacy measures included the proportion of patients who achieved IGA-TS, NRS4, EASI75, a reduction from baseline in the affected body surface area (%BSA), change from baseline in the skin pain NRS score, EASI50 and more, measured at various time points. The study also tracked the frequency, duration and severity of adverse events associated with the use of Opzelura.
For more information on the study, visit https://www.clinicaltrials.gov/study/NCT06238817 .
About Opzelura ® (ruxolitinib) cream
Opzelura (ruxolitinib) cream, a novel cream formulation of Incyte’s selective JAK1/JAK2 inhibitor ruxolitinib, is approved by the US Food & Drug Administration for the topical treatment of nonsegmental vitiligo in patients 12 years of age and older, is the first and only treatment for repigmentation approved for use in the United States.
Opzelura is also approved in the US for the topical short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis (AD) in non-immunocompromised patients 2 years of age and older whose disease is not adequately controlled with topical prescription therapies, or when those therapies are not advisable. Use of Opzelura in combination with therapeutic biologics, other JAK inhibitors, or potent immunosuppressants, such as azathioprine or cyclosporine, is not recommended.
In Europe, Opzelura ® (ruxolitinib) cream 15mg/g is approved for the treatment of non-segmental vitiligo with facial involvement in adults and adolescents from 12 years of age.
Incyte has worldwide rights for the development and commercialization of Opzelura.
About Incyte®
Incyte is redefining what’s possible in biopharmaceutical innovation. Through deep scientific expertise and a relentless focus on patients, we have built an established portfolio of first-in-class medicines and an extensive portfolio of next-generation medicines across our key franchises: Hematology, Oncology, and Inflammation & Autoimmunity.
