Opzelura

Atopic Dermatitis

Opzelura is used for the short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis (eczema). It is typically prescribed for patients who are not immunocompromised and whose condition is not well controlled with other topical prescription therapies. It may also be used when other treatments are not recommended for the patient.

Nonsegmental Vitiligo

This medication is also used to treat nonsegmental vitiligo. In patients with this condition, the cream is applied to affected areas to help restore skin color (repigmentation).

Patient Eligibility

• It is approved for use in adults and children 12 years of age and older.
• It is not intended for use in patients with weakened immune systems.
• It is specifically for topical use on the skin and is not for use in the eyes, mouth, or vagina.

Mechanism of Action

Opzelura contains the active ingredient ruxolitinib, which belongs to a class of medications known as Janus kinase (JAK) inhibitors. It works by blocking the activity of two specific enzymes, JAK1 and JAK2. These enzymes are part of a signaling pathway that helps regulate the immune system and inflammation.

How it Helps Atopic Dermatitis

In conditions like atopic dermatitis (eczema), the immune system becomes overactive. This overactivity sends signals through the JAK pathway that lead to skin inflammation, redness, and intense itching. By blocking these enzymes directly at the site of application, Opzelura helps to:

• Reduce inflammation: It calms the immune response in the skin.
• Relieve itching: It interrupts the signals that cause the urge to scratch.

How it Helps Vitiligo

In nonsegmental vitiligo, the immune system mistakenly attacks and destroys melanocytes, which are the cells that give skin its color. While the exact mechanism is not fully understood, Opzelura is thought to block the specific immune signals that cause this attack. By stopping the immune system from targeting these cells, the medication may allow the skin to regain its natural pigment over time.

Common Side Effects

The most frequent side effects reported by users of Opzelura include:

• Application site reactions, such as itching, redness, or acne
• Common cold symptoms, such as a runny nose or sore throat
• Headache
• Diarrhea
• Fever
• Urinary tract infections
• Inflammation of the hair follicles (folliculitis)

Serious Side Effects

Opzelura belongs to a class of drugs that can cause serious, potentially life-threatening side effects. Contact a healthcare provider immediately if you experience:

• Serious Infections: Signs of infection such as fever, chills, persistent cough, or painful skin rashes, including shingles.
• Blood Clots: Symptoms like sudden leg swelling, chest pain, or difficulty breathing.
• Heart Problems: Including signs of a heart attack or stroke, such as chest pressure, sudden weakness, or slurred speech.
• Malignancies: Development of new skin growths or changes in moles, as well as risks for lymphoma and other cancers.
• Low Blood Cell Counts: Symptoms of anemia or low white blood cell counts, such as unusual tiredness or frequent infections.

This is not a complete list of side effects. Always consult a healthcare provider for medical advice regarding side effects.

Boxed Warning

Opzelura carries a boxed warning regarding serious risks associated with the Janus kinase (JAK) inhibitor drug class. While Opzelura is a topical cream, the following risks observed with oral JAK inhibitors are considered relevant:

• Serious Infections: Users may be at risk for developing serious infections that could lead to hospitalization or death, including tuberculosis and other bacterial, fungal, or viral infections like shingles.
• Mortality: An increased rate of all-cause death, including sudden cardiovascular death, has been observed in certain populations using oral JAK inhibitors.
• Malignancy: There is an increased risk of lymphoma and other cancers, such as lung cancer. Non-melanoma skin cancers have also been reported specifically in patients using Opzelura.
• Major Adverse Cardiovascular Events: Risks include heart attack and stroke, particularly in patients who are current or past smokers or have other cardiovascular risk factors.
• Thrombosis: Serious and sometimes fatal blood clots, including deep vein thrombosis and pulmonary embolism, have occurred.

Additional Precautions

Blood Count Monitoring

Low blood cell counts, such as anemia or low white blood cell levels, may occur. Healthcare providers may monitor blood counts before and during treatment to ensure levels remain within a safe range.

Hypersensitivity

Allergic reactions can occur with the use of this medication. If symptoms of a serious reaction develop, such as hives, swelling, or trouble breathing, use of the medication should be discontinued immediately.

Skin Monitoring

Because non-melanoma skin cancers have been reported, it is recommended to perform regular skin examinations while using this treatment.

Pregnancy

There is limited data on the use of Opzelura in pregnant women to determine if there is a drug-associated risk for major birth defects or miscarriage. In animal studies where ruxolitinib was administered orally, developmental toxicity was observed at doses much higher than the levels typically absorbed through the skin in humans. Because the effects on an unborn baby are not fully known, you should discuss the potential risks and benefits with your healthcare provider if you are pregnant or planning to become pregnant.

A pregnancy registry has been established to monitor the health of individuals who use Opzelura during pregnancy and the outcomes for their infants.

Breastfeeding

It is not known if ruxolitinib passes into human breast milk after topical application. However, because oral ruxolitinib is present in animal milk and has the potential to cause serious adverse reactions in a nursing child, breastfeeding is not recommended during treatment with Opzelura. You should wait at least four weeks after your final dose before beginning or resuming breastfeeding.

Dosage Forms

Opzelura is available in the following form:

• Topical cream

This medication is packaged in tubes for application directly to the skin.

FDA Approval Status

Opzelura (ruxolitinib) cream was first approved by the U.S. Food and Drug Administration (FDA) in September 2021 for atopic dermatitis. In July 2022, the FDA expanded its approval to include the treatment of nonsegmental vitiligo in adults and pediatric patients 12 years of age and older.

Approved Indications

The FDA has authorized Opzelura for the following specific uses:

• Atopic Dermatitis: Short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis in non-immunocompromised patients whose disease is not adequately controlled with other topical prescription therapies or when those therapies are not advisable.
• Nonsegmental Vitiligo: Topical treatment of nonsegmental vitiligo in patients 12 years of age and older.

Boxed Warning

Opzelura includes a Boxed Warning, the most serious warning required by the FDA. Although Opzelura is a topical cream, the warning is based on safety data from oral Janus kinase (JAK) inhibitors. The warning highlights risks including:

• Serious infections that may lead to hospitalization or death, including tuberculosis and bacterial, invasive fungal, or viral infections.
• Higher rate of all-cause mortality.
• Increased risk of malignancies, including lymphomas and lung cancer.
• Major adverse cardiovascular events (MACE), such as heart attack or stroke.
• Thrombosis, including deep vein thrombosis and pulmonary embolism.

Insurance and Prior Authorization

Most health insurance plans, including commercial insurance and Medicare, may cover Opzelura, but they often require prior authorization. This means your healthcare provider must submit documentation to your insurance company explaining why the medication is medically necessary before the plan will agree to pay for it. Your specific out-of-pocket cost will depend on your plan's deductible, copay, or coinsurance levels.

Financial Assistance Programs

Because Opzelura is a specialty medication, the retail price can be high. Several options may be available to help reduce the cost:

• Copay Savings Cards: The manufacturer may offer a savings card for patients with commercial insurance. This can significantly lower the monthly out-of-pocket expense for eligible individuals.
• Patient Assistance: For patients who are uninsured or have limited income, there may be programs that provide the medication at a reduced cost or for free.

Generic Availability

There is currently no generic version of Opzelura available. As a brand-name medication with patent protection, it typically costs more than older topical treatments. The price is also influenced by the size of the tube prescribed, which is commonly 60 grams.

Pharmacy Variations

The cost of Opzelura can vary between different retail and specialty pharmacies. It is often helpful to compare prices or check if your insurance requires you to use a specific specialty pharmacy to fill the prescription.