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OPZELURA is indicated for the topical short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis in non-immunocompromised adult and pediatric patients 2 years of age and older whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable.

Limitations of Use: Use of OPZELURA in combination with therapeutic biologics, other JAK inhibitors, or potent immunosuppressants such as azathioprine or cyclosporine is not recommended.

TRIAL OVERVIEW: AGES 2-11

EVALUATED IN A PHASE 3 TRIAL OF PEDIATRIC PATIENTS1-3

 

330 pediatric patients 2-11 years of age were included in a phase 3, double-blind, randomized, vehicle-controlled trial.1-3

Patients were randomized to monotherapy with OPZELURA, ruxolitinib cream 0.75%, or vehicle BID for 8 weeks and as needed during a 44-week extension.1-3

key inclusion criteria2,3:

  • 2-11 years of age
  • Diagnosis of AD ≥3 months
  • IGA score of 2 or 3*
  • Affected BSA of 3% to 20%
  • Itch NRS ≥4 (among patients aged 6-11 years)
Primary endpoint3
IGA-TSProportion of patients who achieved clear (IGA 0) or almost clear (IGA 1) 
skin with at least a 2-point improvement from baseline

Safety and tolerability assessments included the frequency of reported TEAEs, treatment-related AEs, and AEs leading to discontinuation.2,3

*Severity scale of 0 to 4.1

 

At baseline:

  • Patients had a mean affected BSA of ≈11%3
  • 76% of the patients had an IGA score of 3 (moderate)3

AD, atopic dermatitis; AE, adverse event; BSA, body surface area; IGA, Investigator's Global Assessment; IGA-TS, Investigator’s Global Assessment treatment success; NRS, numerical rating scale; TEAE, treatment-emergent adverse event.

BASELINE PATIENT CHARACTERISTICS1,3

Study Participants (N = 330)
Age (years)
2-651%
7-<1249%
Race
White55%
Black32%
Asian6%
Other7%
Lesion appearance (IGA*)
Score of 224%
Score of 376%
BSA %
Mean10.5%

*Patients had a baseline IGA score of 2 to 3 on a severity scale of 0 to 4.1

BSA, body surface area; IGA, Investigator's Global Assessment.

 
IN THE 44-WEEK EXTENSION PERIOD2
  • Patients initially randomized to OPZELURA in the TRuE-AD3 clinical trial remained on their regimen2
  • Patients initially randomized to vehicle were rerandomized 1:1 to ruxolitinib cream 0.75% or OPZELURA2
  • Patients self-evaluated active AD lesions and treated as needed (up to 20% BSA)2
  • Patients were instructed to stop treatment 3 days after lesion clearance and restart treatment at the first sign of recurrence2
  • If new lesions were extensive or appeared in new areas, patients were instructed to contact the investigator to determine if an unscheduled additional visit was needed. Rescue treatment was not permitted2
  • Study visits occurred every 4 weeks2
  • Patients initially randomized to OPZELURA in the TRuE-AD3 clinical trial remained on their regimen2
  • Patients initially randomized to vehicle were rerandomized 1:1 to ruxolitinib cream 0.75% or OPZELURA2
  • Patients self-evaluated active AD lesions and treated as needed (up to 20% BSA)2
  • Patients were instructed to stop treatment 3 days after lesion clearance and restart treatment at the first sign of recurrence2
  • If new lesions were extensive or appeared in new areas, patients were instructed to contact the investigator to determine if an unscheduled additional visit was needed. Rescue treatment was not permitted2
  • Study visits occurred every 4 weeks2
A diagram showing the trial design for the OPZELURA clinical trial of patients age 2 to 11
A diagram showing the trial design for the OPZELURA clinical trial of patients age 2 to 11

The graphic above shows the study design for OPZELURA, which was studied in a double-blind, randomized, vehicle-controlled trial (TRuE-AD3). The study included 330 pediatric patients 2-11 years of age with an affected BSA of 3% to 20% and IGA score of 2 (24% of subjects) to 3 (76% of subjects) on a severity scale of 0 to 4. Patients were randomized to monotherapy with OPZELURA, ruxolitinib cream 0.75%, or vehicle twice daily for 8 weeks.1
Eligible patients could continue treatment for an additional 44 weeks in an extension period where OPZELURA was applied to AD areas twice daily as needed. Patients were instructed to stop treatment 3 days after lesion clearance and restart at the first sign of recurrence.1,2

AD, atopic dermatitis; BID, twice daily; BSA, body surface area; IGA, Investigator's Global Assessment.

Important Safety Information and indication

 

INDICATION

OPZELURA is indicated for the topical short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis in non-immunocompromised adult and pediatric patients 2 years of age and older whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable.

Limitations of Use: Use of OPZELURA in combination with therapeutic biologics, other JAK inhibitors, or potent immunosuppressants such as azathioprine or cyclosporine is not recommended.

IMPORTANT SAFETY INFORMATION

SERIOUS INFECTIONS
Patients treated with oral Janus kinase (JAK) inhibitors for inflammatory conditions are at risk for developing serious infections that may lead to hospitalization or death. Reported infections include:
  • Active tuberculosis, which may present with pulmonary or extrapulmonary disease.
  • Invasive fungal infections, including cryptococcosis and pneumocystosis.
  • Bacterial, viral, including herpes zoster, and other infections due to opportunistic pathogens.
Avoid use of OPZELURA in patients with an active, serious infection, including localized infections. If a serious infection develops, interrupt OPZELURA until the infection is controlled. Carefully consider the benefits and risks of treatment prior to initiating OPZELURA in patients with chronic or recurrent infection. Closely monitor patients for the development of signs and symptoms of infection during and after treatment with OPZELURA.

Serious lower respiratory tract infections were reported in the clinical development program with topical ruxolitinib.

No cases of active tuberculosis (TB) were reported in clinical trials with OPZELURA. Cases of active TB were reported in clinical trials of oral JAK inhibitors used to treat inflammatory conditions. Consider evaluating patients for latent and active TB infection prior to administration of OPZELURA. During OPZELURA use, monitor patients for the development of signs and symptoms of TB.

Viral reactivation, including cases of herpes virus reactivation (e.g., herpes zoster), were reported in clinical trials with JAK inhibitors used to treat inflammatory conditions including OPZELURA. If a patient develops herpes zoster, consider interrupting OPZELURA treatment until the episode resolves.

Hepatitis B viral load (HBV-DNA titer) increases, with or without associated elevations in alanine aminotransferase and aspartate aminotransferase, have been reported in patients with chronic HBV infections taking oral ruxolitinib. OPZELURA initiation is not recommended in patients with active hepatitis B or hepatitis C.

MORTALITY

In a large, randomized, postmarketing safety study in rheumatoid arthritis (RA) patients 50 years of age and older with at least one cardiovascular risk factor comparing an oral JAK inhibitor to tumor necrosis factor (TNF) blocker treatment, a higher rate of all-cause mortality, including sudden cardiovascular death, was observed with the JAK inhibitor. Consider the benefits and risks for the individual patient prior to initiating or continuing therapy with OPZELURA.

MALIGNANCIES

Malignancies were reported in patients treated with OPZELURA. Lymphoma and other malignancies have been observed in patients receiving JAK inhibitors used to treat inflammatory conditions. In RA patients treated with an oral JAK inhibitor, a higher rate of malignancies (excluding non-melanoma skin cancer (NMSC)) was observed when compared with TNF blockers. Patients who are current or past smokers are at additional increased risk.

Consider the benefits and risks for the individual patient prior to initiating or continuing therapy with OPZELURA, particularly in patients with a known malignancy (other than successfully treated non-melanoma skin cancers), patients who develop a malignancy when on treatment, and patients who are current or past smokers.

Non-melanoma skin cancers, including basal cell and squamous cell carcinoma, have occurred in patients treated with OPZELURA. Perform periodic skin examinations during OPZELURA treatment and following treatment as appropriate. Exposure to sunlight and UV light should be limited by wearing protective clothing and using broad-spectrum sunscreen.

MAJOR ADVERSE CARDIOVASCULAR EVENTS (MACE)

In RA patients 50 years of age and older with at least one cardiovascular risk factor treated with an oral JAK inhibitor, a higher rate of major adverse cardiovascular events (MACE) (defined as cardiovascular death, myocardial infarction, and stroke), was observed when compared with TNF blockers. Patients who are current or past smokers are at additional increased risk. Discontinue OPZELURA in patients who have experienced a myocardial infarction or stroke.

Consider the benefits and risks for the individual patient prior to initiating or continuing therapy with OPZELURA, particularly in patients who are current or past smokers and patients with other cardiovascular risk factors. Patients should be informed about the symptoms of serious cardiovascular events and the steps to take if they occur. Discontinue OPZELURA in patients that have experienced a myocardial infarction or stroke.

THROMBOSIS

Thromboembolic events were observed in trials with OPZELURA. Thrombosis, including pulmonary embolism (PE), deep venous thrombosis (DVT), and arterial thrombosis have been reported in patients receiving JAK inhibitors used to treat inflammatory conditions. Many of these adverse reactions were serious and some resulted in death. In RA patients 50 years of age and older with at least one cardiovascular risk factor treated with an oral JAK inhibitor, a higher rate of thrombosis was observed when compared with TNF blockers. Avoid OPZELURA in patients at risk. If symptoms of thrombosis occur, discontinue OPZELURA and treat appropriately.

CYTOPENIAS

Thrombocytopenia, anemia, neutropenia, lymphopenia, and leukopenia were reported in the clinical trials with OPZELURA. Consider the benefits and risks for individual patients who have a known history of these events prior to initiating therapy with OPZELURA. Perform CBC monitoring as clinically indicated. Discontinue OPZELURA if signs and/or symptoms associated with clinically significant decreases in laboratory values occur.

Lipid Elevations

Treatment with oral ruxolitinib has been associated with increases in lipid parameters including total cholesterol, low-density lipoprotein (LDL) cholesterol, and triglycerides.

Adverse Reactions

In atopic dermatitis, the most common adverse reactions (≥1%) are nasopharyngitis, bronchitis, ear infection, eosinophil count increased, urticaria, diarrhea, folliculitis, tonsillitis, rhinorrhea, upper respiratory tract infection, COVID-19, application site reaction, pyrexia, and white blood cell decreased.

Pregnancy Registry

There is a pregnancy registry that monitors pregnancy outcomes in pregnant persons exposed to OPZELURA during pregnancy. Pregnant persons exposed to OPZELURA and healthcare providers should report OPZELURA exposure by calling 1-855-463-3463 or visiting www.opzelura.pregnancy.incyte.com.

Lactation

Advise women not to breastfeed during treatment with OPZELURA and for approximately four weeks after the last dose (approximately 5-6 elimination half-lives).

Please see Full Prescribing Information, including Boxed Warning, and Medication Guide for OPZELURA.

Indication

OPZELURA is indicated for the topical short-term and non-continuous chronic treatment of mild to moderate atopic

Important Safety Information

Important Safety Information and indication

Serious Infections

Patients treated with oral Janus kinase (JAK) inhibitors for inflammatory conditions are at risk for developing serious infections that may lead to hospitalization or death. Reported infections include:

Patients treated with oral Janus kinase (JAK) inhibitors for inflammatory conditions are at risk for developing serious infections that may lead to hospitalization or death. Reported infections include:

REFERENCES: 1. OPZELURA Prescribing Information. Wilmington, DE: Incyte Corporation. 2. Eichenfield LF, Stein Gold LF, Simpson EL, et al. 52-week safety and disease control with ruxolitinib cream in children aged 2-11 years with atopic dermatitis: results from the phase 3 TRuE-AD3 study. Presented at the European Academy of Dermatology and Venereology Congress; September 25-28, 2024; Amsterdam, Netherlands. 3. Eichenfield LF, Stein Gold LF, Simpson EL, et al. Efficacy and safety of ruxolitinib cream in children aged 2 to 11 years with atopic dermatitis: Results from TRuE-AD3, a phase 3, randomized double-blind study. J Am Acad Dermatol. 2025;93(3):689-698. doi:10.1016/j.jaad.2025.05.1385