The first FDA-approved medical treatment for repigmentation of vitiligo has finally arrived. On July 18th, the U.S. Food and Drug Administration (FDA) approved Opzelura™ (ruxolitinib) cream 1.5% for the topical treatment of vitiligo in patients 12 years of age and older. (Insert applause for this groundbreaking news and history-in-the-making kind of moment for vitiligo and treatment of this condition.)

Why is this big news? If you are familiar with vitiligo treatments, you may know that prior to the FDA’s approval of Opzelura, the only FDA-approved treatment for vitiligo was monobenzone, a drug that removes pigment from the skin. (Yes, you read that correctly—the treatment was typically used to remove remaining pigment in those who had lost a significant amount.)

So how is Opzelura different? Opzelura is the first treatment of its kind for vitiligo in that it has FDA approval for repigmentation of vitiligo. But that’s not all—Opzelura is the only topical formulation of a JAK inhibitor, an emerging treatment with positive results, with FDA approval in the U.S. (Not familiar with JAK inhibitors? We’ve got you covered, keep reading!)

Opzelura was developed by Incyte, a biopharmaceutical company committed to discovering and developing best-in-class medicines to bring innovative solutions to patients in need, including dermatologic conditions.

Here’s your guide to Opzelura and what this announcement means for those with vitiligo—and the future of vitiligo treatments.

What is Opzelura and how does it work?

Opzelura (ruxolitinib) cream 1.5% is the the first and only FDA-approved medical treatment for the topical treatment of vitiligo in patients 12 years of age and older. It’s also the only topical formulation of a Janus kinase (JAK) inhibitor in the U.S. To fully understand how this treatment works, let’s dive into how vitiligo causes depigmentation—and how Opzelura works to repigment the skin.

We’ll start with the basics. You may already know that vitiligo is an autoimmune disease, which occurs when the body attacks and damages its own cells due to over-activity of the immune system. In vitiligo, immune cells attack melanocytes, which produce melanin, the skin’s pigment. JAK proteins are responsible for sending the signals that tell the immune system to attack melanocytes. Overactivity of signals from JAK proteins are believed to drive the inflammation that is involved in the development and progression of vitiligo.

So how does Opzelura fit in? Opzelura is a ruxolitinib cream, which is a JAK inhibitor—and inhibitors prevent JAK proteins from sending the signals that tell the immune system to attack melanocytes. By stopping the attack of melanocytes, JAK inhibitors give the body the ability to repigment the skin.

How are JAK inhibitors different from previous vitiligo treatments? JAK inhibitors are more targeted compared to other vitiligo treatments in that they focus on stopping one part of the immune system without shutting it off entirely. As a result, the body is still able to fight off infections while the part of the immune system that causes vitiligo is shut off.

How effective is Opzelura in repigmenting vitiligo?

So—how effective is Opzelura in repigmenting vitiligo? Here’s what we know about the positive results of Opzelura in repigmenting vitiligo today.

The FDA’s approval of Opzelura is based on positive results from the TRuE-V clinical trial program, which includes two Phase 3 studies, TRuE-V1 and TRuE-V2. (New to clinical trials? A Phase 3 clinical trial specifically tests new treatments in a larger number of patients, typically to get FDA approval.)

Both studies evaluated the treatment’s safety and efficacy in people with vitiligo. Each study enrolled approximately 300 patients 12 years of age and older with non-segmental vitiligo. (Non-segmental vitiligo is a type of vitiligo that appears on both sides of the body.)

Findings from the studies showed that treatment with 1.5% Opzelura (ruxolitinib) twice daily resulted in significant improvements in facial and total body repigmentation at 24 weeks compared to a non-medicated cream. At week 24, approximately 30% of patients applying ruxolitinib cream achieved 75% or greater improvement in facial vitiligo. More than 15% of patients applying ruxolitinib cream achieved ≥90% improvement from the baseline. See Incyte’s press release for additional details.

What about side effects? According to the FDA, the “most common adverse reactions associated with Opzelura are application site acne, application site itching, common cold, headache, urinary tract infection, application site redness, and fever.” You can find full safety information about Opzelura here. Additionally, Opzelura has a “boxed warning,” which means that there could be a small risk for serious infections, heart disease, blood clotting, cancer, or even death. However, as Dr. John Harris explains in his blog post about the FDA announcement, “these were not seen in the clinical trials for the topical drug ruxolitinib but have been reported in small numbers for those taking the oral version of the drug.” In other words, this is “a ‘better safe than sorry’ approach by the FDA, so that users are made fully aware.”

Read More: “Opzelura, the first FDA-approved drug to treat vitiligo!” by Dr. John Harris, Director of the University of Massachusetts Vitiligo Clinic and Research Center and vitiligo expert

Frequently asked questions about Opzelura

You likely have questions about Opzelura—and we’ve rounded up answers to help you learn as much as possible about this new vitiligo treatment. (Have a question that isn’t addressed here? Visit www.Opzelura.com.)

Is Opzelura available for treatment of vitiligo now?

Yes, Opzelura is currently available to patients in the United States for the treatment of vitiligo. (Heard of Opzelura before? You may have—Opzelura was approved by the FDA for the treatment of eczema in September 2021.)

How do you use Opzelura?

Opzelura should be used as directed by your healthcare provider. Opzelura is a topical cream that is applied twice daily directly to the areas of the skin where there is depigmentation caused by vitiligo. 

How long does Opzelura take to repigment vitiligo?

The TRuE-V clinical trial program included two phase three clinical trials. Results from both clinical trials showed that treatment with Opzelura resulted in significant improvements in facial and total body repigmentation at 24 weeks compared to a non-medicated cream.

Is Opzelura only available in the United States?

As of October 2021, the European Medicines Agency (EMA) has validated the European Marketing Authorization Application (MAA) for Opzelura, indicating that the submission is ready to enter a formal review process. Learn more about the latest on the EMA’s validation.

Why FDA approval of Opzelura matters

This is exciting, right? Yes. (Let’s try that again—YES!!) The FDA’s approval of a treatment for repigmentation of vitiligo is a major milestone for the vitiligo community and the history of vitiligo treatment.

Let’s take a look back at where we were before this announcement. Previously, the only FDA-approved treatment for vitiligo was monobenzone, a drug that removes pigment from the skin. And while Opzelura and JAK inhibitors have been previously approved by the FDA, it was for other diseases and conditions.

When Opzelura was submitted for review by the FDA, the organization granted priority review, which is reserved for medicines that may offer a major advance in treatment where none currently exists. If you ask the vitiligo community, this was absolutely an unmet need—and the FDA agreed.

Opzelura is the first treatment of its kind for vitiligo and is the only topical formulation of a JAK inhibitor approved in the U.S. But this isn’t just a new treatment. This is hope.

For many living with vitiligo, the lack of control over your skin and resulting implications for your image and identity can be devastating. And if the emotional and mental toll isn’t enough, the social stigma can cripple relationships, careers and more. With all of that at stake, what do you do when the options to do something about it are limited? You do all you can—anxiously wait and watch for your skin to lose its pigment, spot by spot.

But not anymore.

This news offers hope for those who want to have an option in how they live (or don’t) with this condition. This news offers hope for the dermatologists who have patients desperately asking for answers after a difficult diagnosis. This news offers hope for the future of vitiligo as an autoimmune disease and the ability to treat it. This news offers hope—and it is undoubtedly the biggest progress the vitiligo community has seen in treatments to date.

Read More

Visit www.Opzelura.com

Read Incyte’s Press Release

Read the Business Wire Press Release

Read Dr. John Harris’ Blog Post (Hear straight from a leading vitiligo expert and dermatologist about why this matters!)