Exploring Unmet Needs and Pipeline Treatments for Vitiligo


Dr Rosmarin navigates unmet needs and the trajectory of vitiligo treatment.

David Rosmarin, MD: Ruxolitinib cream is a big step forward in the treatment of vitiligo, but there are still patients who don’t respond. There’s a lot we don’t know. For example, why are some patients very early responders, some patients late responders, and some nonresponders? It would be helpful to figure that out. And we still need other treatments. Oral JAK inhibitors are important for patients who have progressive vitiligo that you can’t chase with the cream if they’re getting a lot of new spots, and for patients who have a large surface area.

Ruxolitinib cream is limited on its label to 10% or less because that’s how the phase 3 [trial] was designed. But in the phase 2 [trial] for vitiligo and the phase 3 [trial] for atopic dermatitis, up to 20% of the body was used, and that still may be safer than oral treatments. Using the cream on a larger surface area can sometimes make sense, and having studies to show that would be helpful. The synergy between phototherapy and ruxolitinib cream would be helpful as well. Right now, we have limited data for that. We can’t definitively say that the phototherapy plus ruxolitinib cream is better than ruxolitinib cream alone. Further studies into that would be helpful.

We also have a knowledge gap in terms of maintenance. When patients get their pigment back, what’s the best way to help these patients? Should they be using the cream twice a week for maintenance, as we do with topical tacrolimus? Or should they be using it more regularly? Or can they take a holiday for several months? We have to figure that out, and we will with time.

Additionally, patients aren’t truly cured of their vitiligo. It can always come back at any time. It’s a chronic disease. There are other treatments in development, including an antibody that blocks interleukin-15 that may give a long-term remission, but that’s still being evaluated. There are also other treatments that we need to help stimulate the pigment back. What about patients who have vitiligo on their hands and feet, which are challenging-to-treat areas? We still need other treatments for them. We’ve come a long way, but we still have a long way to go.

When I look at the pipeline for vitiligo treatment, ruxolitinib cream is the biggest step that I anticipate in the next five years. I believe it will be increasingly used for patients who have this disease. Some of the other treatments in the pipeline can help as well. Oral JAK inhibitors are in development, and those will likely be next up for approval. Those will help patients who have a larger body surface area or maybe progressive disease. We also have to see if the oral medicines will help patients who fail the topical. There are obviously safety differences, with the orals carrying more systemic adverse effects than the topical. I believe that most patients will go for the topical first, but there are patients who may go for an oral in the first line, and those who fail the topical and then go for an oral agent.

Also in development is a phytoceutical of a plant in Brazil. We still don’t have any data on that, but perhaps that can help patients repigment. There’s also an anti–IL-15 antibody that perhaps can help patients with vitiligo, particularly with a longer-term remission. But the treatments for vitiligo are promising. Now that we have a pathway and a proof of concept that it’s possible to treat the disease, there will be increasing interest. Those are the most likely treatments along the horizon in the next 5 years, but there will be more.

Lastly, there are devices in development, such as the RECELL device, which is used for burns and wound healing. That helps take patients’ pigment cells from elsewhere on the body and transfer them to the areas of vitiligo. This melanocyte transfer technique is done only by a few places in the country, but having this device may make it more accessible to community dermatologists.

In summary, vitiligo is a medical disease, not a cosmetic condition. It’s important for us to be thoughtful about how we treat and approach patients. There’s a lot of learning that providers and payers need to do, and [we need to] work together to make sure we have treatment algorithms that make sense. We want treatments available and accessible when they’re going to have impact on patients and add value. It’s also reasonable to try alternatives to our more expensive options if those are proven to be effective. But we don’t want steps for therapies that are futile. We have a bright future for the treatment of vitiligo, and we’re going to help a lot of patients with these new treatments.

Transcript edited for clarity.

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