FDA Approves First Topical Treatment for Vitiligo: What to Know About Opzelura

For people with vitiligo, a new topical cream is now available. The US Food and Drug Administration (FDA) has approved a topical cream — Opzelura — to treat non-segmental vitiligo, according to a statement by the manufacturer of the drug, Incyte.

Vitiligo is a chronic skin condition where pale white patches develop. It's caused by the loss of melanocytes, the cells that give skin its color. The patches can occur anywhere on the body but commonly on the face, neck, hands, and in skin creases. In some cases, they can cover large areas.

There is no cure for vitiligo, but treatments like Opzelura may help improve the skin's appearance and restore pigment.

Continue reading to learn more about Opzelura, how it works, and what to expect from this topical treatment.

What is vitiligo?

Around 1.9 and 2.8 million adults in the United States have vitiligo. This chronic condition causes the skin to lose color in patches, which often increase in size over time.

The two main types of vitiligo are:

  • Non-segmental or generalized vitiligo – This is the most common form of vitiligo. It typically results in pale patches on both sides of the body, such as both hands. The loss of color may happen quickly and affect a large area.
  • Segmental vitiligo — This form of vitiligo is much less common and only affects one side of the body. It often affects young children, progresses for 1 to 2 years, and then stops.

Rarely, vitiligo affects the whole body. This is known as universal or complete vitiligo.

Vitiligo happens when pigment-producing cells called melanocytes die or stop producing pigments.

Doctors are not entirely sure why it happens, but it may be related to:

  • an autoimmune condition where the immune cells destroy melanocytes
  • a family history
  • a trigger event, such as stress or skin trauma

Anyone of any age can develop vitiligo. Often, symptoms appear before age 20 and may start in early childhood. It's also more common if you have an autoimmune disease such as psoriasis, type 1 diabetes, rheumatoid arthritis, or systemic lupus erythematosus.

What is Opzelura?

Opzelura — active ingredient ruxolitinib 1.5% — was approved by the FDA in September 2021 for mild to moderate eczema (atopic dermatitis). It has now been approved for non-segmental vitiligo in adults and children 12 and older.

It's a selective Janus kinase (JAK) inhibitor that works by blocking two enzymes called JAK1 and JAK2. These enzymes are involved in several chemical pathways that contribute to inflammation.

Doctors typically prescribe oral JAK inhibitors for inflammatory conditions such as rheumatoid arthritis. Opzelura is the only topical JAK inhibitor approved in the US.

Why was it approved?

The approval was based on results from the TRuE-V clinical trial program that included two Phase 3 studies — TRuE-V1 and TRuE-V2. Researchers assessed the safety and efficacy of Opzelura compared to a non-medicated cream in over 600 participants aged 12 and above, with non-segmental vitiligo covering less than 10% of their bodies.

They used a tool called the Vitiligo Area Scoring Index (VASI) to gauge the severity of the vitiligo and measure skin re-pigmentation on the face and body.

At 24 weeks, around 30% of people using the 1.5% ruxolitinib cream twice daily (BID) had significant improvements of at least 75% in their VASI scores. By comparison, only 8% of those treated with the vehicle cream in TRuE-V1 and 13% in TRuE-V2 showed an improvement.

Furthermore, more than 15% of those in the Opzelura group achieved over 90% improvement from baseline compared to 2% in the vehicle cream group.

Further data from a 28-week treatment extension showed that the cream continued its positive effects at 52 weeks.

Were there any side effects?

The safety data from the trial also looked good, with only minor side effects reported. Up to 15% of the ruxolitinib users noted localized acne bumps. But most people were not concerned with these issues as their vitiligo improved, and the bumps disappeared when they stopped the treatment.

Other side effects included:

  • Itching
  • Headache
  • Urinary tract infection
  • Swelling of the skin
  • Fever
  • Nasal cavity inflammation

Why is there a black box warning?

The FDA has placed a black box warning on Opzelura. This is the most serious type of warning, meaning the medication may not be suitable for everyone.

The warning is due to data that shows oral JAK inhibitors cause a small increase in the risk of serious infections, severe heart issues, thrombosis or blood clots, and cancer.

However, using a topical JAK inhibitor differs from the oral form of the drug, and Opzelura contains a far lower concentration of ruxolitinib. In the clinical trials of ruxolitinib, the researchers did not observe any of these risks, but the FDA is being cautious by including the black box warning.

If you're considering using Opzelura, having a conversation with your doctor can help you decide if the benefits outweigh any potential risks.

How do I use Opzelura?

For non-segmental vitiligo, apply a thin layer of Opzelura cream to the affected areas twice daily. You shouldn't use the cream on more than 10% of your skin, and it's important to avoid your eyes, mouth, and vagina.

If you have any form of localized skin infection, you shouldn't use the cream unless you check with your doctor first. It's also a good idea to check with your doctor if you can safely use Opzelura while taking other medications.


The US Food and Drug Administration has now approved Opzelura for non-segmental vitiligo. This topical cream contains the active ingredient ruxolitinib 1.5%. It is a JAK inhibitor that helps reduce inflammation and repigment skin.

Clinical trials have shown that Opzelura is safe and effective, but the FDA has placed a black box warning on the medication. This is because oral JAK inhibitors increase the risk of some serious health conditions. However, Opzelura contains a far lower concentration of ruxolitinib.

Anyone interested in taking Opzelura should discuss the benefits and potential risks with their doctor.

Key takeaways

The FDA has now approved topical Opzelura to treat non-segmental vitiligo.

Vitiligo is a skin condition that results in loss of pigmentation.

Ruxolitinib 1.5% is the active ingredient in Opzelura, a JAK inhibitor that helps reduce inflammation.


Business Wire. Incyte Announces U.S. FDA Approval of Opzelura™ (ruxolitinib) Cream, a Topical JAK Inhibitor, for the Treatment of Atopic Dermatitis (AD)

Incyte. Incyte Announces U.S. FDA Approval Of Opzelura™ (Ruxolitinib) Cream For The Treatment Of Vitiligo

JAMA Dermatology. Prevalence of Vitiligo Among Adults in the United States

NIH. Vitiligo

Opzelura. Prescribing information

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