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"Speaking of Vitiligo..."

Opzelura, the first FDA-approved drug to treat vitiligo!

Monday, July 18, 2022

This is a momentous occasion! One for which we have been waiting for thousands of years (literally). Vitiligo was recognized and described in ancient medical texts from India, called the Rigveda and Atharvaveda and written in around 1400 B.C. At that time, they recommended sunlight and chewing on bavachee seeds as a treatment for vitiligo, and we now know that those seeds contained a chemical called psoralen, which forms a key ingredient in PUVA therapy. This approach was rediscovered in the 1940s and used to treat our patients until narrow band UVB took its place. But I digress, the key message is that vitiligo treatments haven’t changed much in about 3400 years.

Another point to make before we get into the exciting part is that there IS an FDA-approved treatment for vitiligo already – it’s called monobenzyl ether of hydroquinone, or monobenzone. It has been used for many decades, but the problem is that this treatment makes vitiligo WORSE, not better! You may have heard of it as the “depigmenting cream”, or “bleaching cream” that turns the skin of vitiligo patients white after use for about 1-2 years. In fact, Michael Jackson used this cream to treat his vitiligo. But we don’t use this treatment for most patients, since they typically want to make their skin color come back, not go away.

So here it is! Topical ruxolitinib (generic name), or Opzelura (brand name), has just been approved for the treatment of vitiligo by the FDA. I started writing about this drug in 2015, when we were the first to give it to a vitiligo patient orally and it worked. Then, in 2017 Dr. David Rosmarin at Tufts had it put into a cream and he showed it worked topically for patients with vitiligo on their face. Then in 2019 I wrote about the successful results of our Phase 2 clinical trial to test topical ruxolitinib after 6 months, which was tested in 157 volunteers and had great results. Later that year, I wrote about the even better results after 1 year of treatment and we published that study in the journal The Lancet. In 2021 I announced that the results of the larger, Phase 3 study confirmed the previous results and that we were submitting the application to the FDA for approval to be the first approved drug to reverse vitiligo. I mentioned that it can take a year for the FDA to review the application and make a decision, which would have been earlier this spring. But they required more time, announcing a 3-month extension, and so the timeline moved to July 2022, and here we are!

Opzelura was already approved last September, 2021, for a disease called atopic dermatitis, or eczema, so you may have already seen it in the pharmacy. But most couldn’t get it covered by insurance for vitiligo because it wasn’t approved for it, and so NOW IT IS! The Phase 3 clinical trial enrolled volunteers as young as 12 years of age, and so the approval covers its use for kids, 12 years and older. Safety data from the trial looked good – the only side effect noted in those using the drug more than placebo (and therefore attributable to the drug) was localized acne bumps, in about 10-15% of users. So, keep that in mind, but most users were ok with this because their vitiligo was getting better and the bumps went away when they stopped the treatment.

However, please note – you’ll notice on the box that Opzelura has a “black box warning” on the label, meaning that the FDA wants you to know that there could be a small risk for serious infections, heart disease, blood clotting, cancer, or even death. 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, which results in very large concentrations in the blood. These concentrations were NOT seen in trials when volunteers used the cream, but this is a “better safe than sorry” approach by the FDA, so that users are made fully aware.

So, there you have it. Happy “First FDA-approved drug for vitiligo day”! I’m really excited about this and expect this is only the tip of the iceberg – so many more ideas being tested now and I anticipate more options will be available for patients soon. I have my eye on a systemic treatment that I’m hoping will be easier to use and long-lasting, even after it’s discontinued. I’ll provide an update on that later, but for now, this is HUGE!!!