Ruxolitinib cream is the first FDA-approved JAK inhibitor for skin repigmentation in people with vitiligo, paving the way for additional oral and topical JAK inhibitors to treat this autoimmune disorder.
The FDA approved a topical Janus kinase (JAK) inhibitor to treat a skin condition known as vitiligo. The new drug, known as ruxolitinib cream or by its brand name Opzelura, is the first and only FDA-approved topical JAK inhibitor for treating vitiligo in people ages 12 and older.
Experts think that vitiligo occurs when the body’s immune system attacks melanocytes — the cells responsible for producing skin pigment. The only symptom of vitiligo is light-toned patches or spots on the skin. However, the condition can also have an effect on people’s mental health.
JAK inhibitors are medications that block signaling pathways in the body that are responsible for an atypical immune response.
Most of these JAK inhibitors come in pill form, but Opzelura is a topical treatment, which means that a person can apply it directly to their skin.
Findings from a clinical program known as the TRuE-V program prompted the FDA to approve ruxolitinib. The two phase 3 trials included more than 600 adults and teens with vitiligo. The participant population was as follows:
- 53% female participants
- 82% white participants
- 5% Black participants
- 4% Asian participants
Findings
After 24 weeks of applying the cream twice per day, about 30% of participants in both trials saw at least a 75% improvement in facial patches, compared with about 10% of those who applied a placebo cream. About 15% of people treated with ruxolitinib noticed their facial patches improved by more than 90%.
After a year, about 50% of the people taking ruxolitinib experienced at least a 75% improvement in facial patches.
Researchers determined the improvements using the Vitiligo Area Scoring Index, a tool they developed to measure vitiligo severity.
Currently, ruxolitinib cream is only FDA-approved to treat nonsegmental vitiligo, which is the most common type of vitiligo. In nonsegmental vitiligo, patches tend to appear on both sides of the body.
Experts need to conduct research on how ruxolitinib cream affects people with segmental vitiligo, in which light patches appear on just one side or part of the body.
People most often reported mild side effects in the clinical trials for ruxolitinib cream, including:
- acne
- redness
- itchiness at the application site
- throat and nasal inflammation
- headache
- urinary tract infection
- fever
Like other JAK inhibitors, Opzelura’s FDA-approved label includes a boxed warning for:
- serious infections
- heart-related events
- blood clots
- cancer
- death
JAK inhibitors suppress the body’s immune system and can make it difficult for the immune system to clear infections. Experts recommend that people with an active, serious infection avoid using ruxolitinib cream.
In rare cases, people reported serious side effects with the use of some oral JAK inhibitors, but the FDA included these potential risks as a
With ruxolitinib’s approval comes hope for the effective treatment of a condition that previously had few options. Traditionally, people with vitiligo received the following treatments:
- phototherapy administered in a doctor’s office
- topical corticosteroids
- topical calcineurin inhibitors, which had
inconsistent results
Experts performed past research examining oral JAK inhibitors as a treatment for vitiligo. For example, a
The manufacturer of tofacitinib is also studying a topical formulation. However, experts need to perform more clinical trials to determine if the topical formulation is effective at treating vitiligo.
Next in the pipeline may be ritlecitinib, an oral JAK inhibitor that showed promise in a Phase 2b clinical trial.
Other oral and topical JAK inhibitors with clinical proof-of-concept studies include:
Although experts might prefer topical treatments due to fewer safety concerns, an oral option may be better for people whose vitiligo covers a large body surface area. Currently, experts recommend that people only apply ruxolitinib cream to 10% or less of the body’s surface area.
Other agents that researchers are studying in ongoing clinical trials
Dermatologists hope to eventually find a product that can offer total skin repigmentation, along with a manageable safety profile.
As the first topical JAK inhibitor, experts consider ruxolitinib cream a breakthrough in managing vitiligo, which previously had few treatment options.
Due to pricing and insurance coverage, doctors may only prescribe ruxolitinib cream after other treatments, such as phototherapy and corticosteroids, ineffectively manage symptoms. People need to keep in mind that it can take 24 weeks or longer to see satisfactory results with ruxolitinib cream.
Ruxolitinib may not work for everyone, but the approval represents important progress in understanding the underlying course of the condition, paving the way for additional treatment options for vitiligo.