The Food and Drug Administration has approved a first-of-its-kind treatment for vitiligo, a condition that causes patches of discoloration on the skin and, currently, has few other therapeutic options.
The treatment, known as ruxolitinib, is now the only one approved in the U.S. to repigment skin in people with the most common, “nonsegmental” form of vitiligo. Incyte, the Delaware-based company that developed ruxolitinib, said the FDA based its decision on two clinical trials that each enrolled a few hundred participants and found the drug significantly improved repigmentation in the face and body.
For the treatment of vitiligo, Incyte formulated ruxolitinib as a topical cream, which it plans to sell under the brand name Opzelura. An oral version of the drug, called Jakafi, has been on the U.S. market for more than a decade, approved for use in a couple bone marrow diseases as well as a condition that can occur after a patient receives a stem cell transplant.
Light therapy and ointments meant for eczema are commonly used to treat vitiligo. But healthcare professionals argue that more options are needed. In a statement provided by Incyte, David Rosmarin, vice chair of research and education in the Tufts Medical Center’s dermatology department, called vitiligo “unpredictable” and “particularly difficult to treat.”
“There have been no FDA-approved therapies available to date and the approval of Opzelura therefore marks a significant milestone,” said Rosmarin, who’s also been involved with studies evaluating ruxolitinib’s effectiveness against vitiligo.
In December, the FDA accepted Incyte’s application for Opzelura and agreed to give it an expedited review. A verdict was expected by April — but in mid-March, Incyte disclosed the agency needed more time to conduct its review and set a new decision deadline of July 18.
Brian Abrahams, an analyst at the investment firm RBC Capital Markets, wrote in a note to clients Monday that his team believed some concerns had arose after this delay. That’s partially because ruxolitinib blocks a type of immune system-regulating protein known as a JAK, and, in recent years, drug agencies have taken issue with the safety of JAK inhibitors developed by Pfizer, Eli Lilly, and others.
The worry among investors, according to Abrahams, was that even though Opzelura appeared safe and effective against vitiligo, the FDA “might be overly cautious in a more cosmetically-oriented indication given their historical scrutiny” of JAK drugs.
Still, Abrahams wrote that Wall Street generally expected approval. With that now in hand, the RBC team expects the approval will add more than $300 million in annual Opzelura sales, providing an “important step” toward the drug becoming a blockbuster product. Last September, the FDA handed Opzelura its first-ever approval, clearing the cream as a treatment for eczema.
Incyte’s share price was up 2%, to around $80.50, in late Tuesday morning trading.