Opzelura (ruxolitinib) - 2 Indications

Scroll down for more information on each indication:

• for mild to moderate atopic dermatitis in patients 12 years of age and older; approved September of 2021
• for the treatment of nonsegmental vitiligo in patients 12 years of age and older; approved July 2022

General Information

Opzelura is a topical cream formulation of ruxolitinib, a Janus kinase (JAK) inhibitor. 

Opzelura is specifically indicated for:

• the topical short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis 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.
• for the topical treatment of nonsegmental vitiligo in adult and pediatric patients 12 years of age and older

Opzelura is supplied as a cream for topical use. Scroll down for dosing/recommendations for each indication.

Mechanism of Action

Opzelura is a topical cream formulation of ruxolitinib, a Janus kinase (JAK) inhibitor. It inhibits JAK1 and JAK2 which mediate the signaling of a number of cytokines and growth factors that are important for hematopoiesis and immune function. JAK signaling involves recruitment of STATs (signal transducers and activators of transcription) to cytokine receptors, activation and subsequent localization of STATs to the nucleus leading to modulation of gene expression. The relevance of inhibition of specific JAK enzymes to therapeutic effectiveness is not currently known.

Side Effects

Adverse effects associated with the use of Opzelura may include, but are not limited to, the following:

• nasopharyngitis
• diarrhea
• bronchitis
• ear infection
• eosinophil count increased
• urticaria
• folliculitis
• tonsillitis
• rhinorrhea

The Opzelura drug label comes with the following Black Box Warning: Serious infections leading to hospitalization or death, including tuberculosis and bacterial, invasive fungal, viral, and other opportunistic infections, have occurred in patients receiving Janus kinase inhibitors for inflammatory conditions. Higher rate of all-cause mortality, including sudden cardiovascular death have been observed in patients treated with Janus kinase inhibitors for inflammatory conditions. Lymphoma and other malignancies have been observed in patients treated with Janus kinase inhibitors for inflammatory conditions. Higher rate of MACE (including cardiovascular death, myocardial infarction, and stroke) has been observed in patients treated with Janus kinase inhibitors for inflammatory conditions. Thrombosis, including deep venous thrombosis, pulmonary embolism, and arterial thrombosis, some fatal, have occurred in patients treated with Janus kinase inhibitors for inflammatory conditions.

Indication 1 - the topical treatment of mild to moderate atopic dermatitis 

Dosing/Administration

Instruct patients to apply a thin layer of OPZELURA twice daily to affected areas of up to 20% body surface area. Stop using when signs and symptoms (e.g., itch, rash, and redness) of atopic dermatitis resolve. If signs and symptoms do not improve within 8 weeks, patients should be re-examined by their healthcare provide

Clinical Trial Results

The FDA approval was based on data from the TRuE-AD (Topical Ruxolitinib Evaluation in Atopic Dermatitis) clinical trial program, consisting of two randomized, double-blind, vehicle-controlled Phase 3 studies (TRuE-AD1 and TRuE-AD 2) evaluating the safety and efficacy of Opzelura in more than 1,200 adolescents and adults with mild to moderate AD. Results from the studies showed patients experienced significantly clearer skin and itch reduction when treated with Opzelura cream 1.5% twice daily (BID), compared to vehicle (non-medicated cream). Significantly more patients treated with Opzelura achieved Investigator’s Global Assessment (IGA) Treatment Success (IGA-TS, primary endpoint) at Week 8 (defined as an IGA score of 0 [clear] or 1 [almost clear] with at least a 2-point improvement from baseline): 53.8% in TRuE-AD1 and 51.3% in TRuE-AD2, compared to vehicle (15.1% in TRuE-AD1, 7.6% in TRuE-AD2. Significantly more patients treated with Opzelura experienced a clinically meaningful reduction in itch from baseline at Week 8, as measured by a ≥4-point reduction in the itch Numerical Rating Scale (itch NRS4): 52.2% in TRuE-AD1 and 50.7% in TRuE-AD2, compared to vehicle (15.4% in TRuE-AD1, 16.3% in TRuE-AD2), among patients with an NRS score of at least 4 at baseline.

Indication 2 - the topical treatment of nonsegmental vitiligo in adult and pediatric patients 12 years of age and older

Dosing/Administration

Instruct patients to apply a thin layer of Opzelura twice daily to affected areas of up to 10% body surface area. Satisfactory patient response may require treatment with Opzelura for more than 24 weeks. If the patient does not find the repigmentation meaningful by 24 weeks, the patient should be re-evaluated by the healthcare provider.

Clinical Trial Results

FDA was based on data from the pivotal Phase 3 TRuE-V clinical trial program (TRuE-V1 and TRuE-V2), evaluating the safety and efficacy of Opzelura versus vehicle in more than 600 people with nonsegmental vitiligo, age 12 and older. 

Results at Week 24, which were consistent across both studies, showed that approximately 30% of patients treated with Opzelura achieved ≥75% improvement from baseline in the facial Vitiligo Area Scoring Index (F-VASI75), the primary endpoint, compared to approximately 8% and 13% of patients treated with vehicle in TRuE-V1 and TRuE-V2, respectively. At Week 52, approximately 50% of Opzelura-treated patients achieved F-VASI75.

Additionally, at Week 24, more than 15% of patients treated with Opzelura achieved ≥90% improvement from baseline in F-VASI (F-VASI90), compared to approximately 2% of patients treated with vehicle. At Week 52, the percentage of Opzelura-treated patients who achieved F-VASI90 doubled to approximately 30%.