Awaiting FDA Decision on Ruxolitinib Cream for Vitiligo

The US Food and Drug Administration (FDA) has delayed decision on the supplemental New Drug Application (sNDA) for ruxolitinib cream as a potential treatment for patients with vitiligo, extending the anticipated decision date from mid-April to July 2022.

The extended wait was announced in close alignment with the beginning of the American Academy of Dermatology (AAD) 2022 Annual Meeting in Boston, where anticipated data supporting the topical JAK inhibitor’s benefit in treating vitiligo will be shared and widely discussed over the weekend.

In the second segment of an interview at AAD 2022, prominent vitiligo researcher John E. Harris, MD, PhD, professor and chair of the Department of Dermatology at UMass Chan Medical School, explained how the FDA is further assessing safety outcomes with ruxolitinib prior to their decision.

“The drug has already been approved for atopic dermatitis, so the assumption would be, ‘Well they already approved it, so what do you need to review at this point?’,” Harris said. “There’s always a question whether patients with different diseases will react differently to the same drug, and sometimes the FDA wants to consider what’s the disease that we’re targeting, and balance the benefit with the potential safety issues and do that with each disease.”

“I think that they are prioritizing safety in their review, and we appreciate that—I think that’s a good thing,” he added.

Harris also discussed what more he is interested in learning about ruxolitinib as a vitiligo therapy in future real-world assessments.

Key among those outcomes are the therapy’s durability in vitiligo management, its long-term impact on patients’ disease severity, its association with treatment-related adverse events including acne, any potential risks of long-term systemic absorption, and just how much surface area vitiligo patients may be willing to use the topical therapy on.

“If they have really widespread disease, then the topical usually isn’t sufficient to treat the entire body,” Harris explained. “We think about 5-10% is the limit for that.”

Regardless the timeline for the would-be vitiligo drug, Harris and colleagues have piqued interest in the future of care.

“It’s a lot of questions, but they’re really good questions, exciting questions to ask,” he said. “I can’t wait to see the results because people are going to get better, and that’s what we care about.”