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FDA Approved Opzelura — With Some Strings Attached


Incyte will be required to conduct postmarketing study and set up patient registry to monitor whether the topical JAK inhibitor has adverse effects on mothers, fetuses and infants.

Although the FDA has warned about serious heart-related adverse events related to oral therapies in the Janus kinase (JAK) inhibitor class, it recently approved the first topical version to treat atopic dermatitis. Approved in September, Incyte’s Opzelura (ruxolitinib) cream is for the short-term treatment of mild-to-moderate atopic dermatitis in patients 12 years of age.

The approval was based on two studies (TRuE-AD1 and TRuE-AD2) that evaluated Opzelura in more than 1,200 adolescents and adults with mild to moderate atopic dermatitis. Results from these studies showed patients experienced clearer skin and itch reduction when treated with Opzelura cream twice daily compared with a non-medicated cream.

Atopic dermatitis is a common, chronic skin condition with persistent or relapsing lesions. It occurs in 7.3% of adults in the United States, and the prevalence is increasing. Of those affected with the disease, about 40% have moderate or severe symptoms. Many patients also suffer from asthma and allergic rhinitis along with intense itching and skin infection.

Research shows dysregulation of the JAK-STAT pathway contributes to key features of atopic dermatitis such as itch and inflammation. Opzelura targets the JAK1/JAK2 pathway.

In June, the FDA had extended the review period for Opzelura, as it did with all JAK inhibitors as a result of data released by Pfizer of a postmarketing study of cardiovascular safety trial of its JAK inhibitor Xeljanz (tofacitinib). Investigators found that the use of JAK inhibitors appears to be associated with major cardiovascular adverse events and malignancies.

As a result of an extended review, the regulatory agency required warnings on the JAK inhibitors approved to treat arthritis and ulcerative colitis about the increased risk of serious heart-related events such as heart attack or stroke, cancer, blood clots, and death.

Opzelura’s label also includes a warning about cardiovascular risks of JAK inhibitors taken by mouth. The FDA has also required Incyte to conduct postmarketing studies, including in patients 12 to 18 years of age, and a pregnancy exposure registry to monitor the treatment for any maternal, fetal and infant adverse effects.

Incyte, which is headquartered in Wilmington, Delaware, is also studying Opzelura as a treatment for vitiligo, an autoimmune disease that results in loss of pigmentation of the skin. The company recently announced results of a phase 3 trial in patients 12 years of age and older. An analysis at week 24 showed treatment with Opzelura twice daily resulted in improvement in primary and secondary end points. Treatment-emergent adverse events were consistent with previous studies, with no serious adverse events reported.

Overactivity of the JAK signaling pathway has been shown to drive inflammation involved in vitiligo. It affects about 0.5% to 2.0% of the population globally, and there are no FDA-approved drug therapies available.

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