The U.S. Food and Drug Administration (FDA) has approved Tepezza (teprotumumab-trbw) for the treatment of adults with thyroid eye disease. This is the first drug approved for this rare condition.
Thyroid eye disease is a common problem that develops in people with Graves’ disease. Thyroid eye disease is associated with the outward bulging of the eye that can cause a variety of symptoms such as eye pain, double vision, light sensitivity, and/or difficulty closing the eye. This disease impacts a relatively small number of Americans and tends to be more predominant in women than men. Although this condition impacts relatively few individuals, thyroid eye disease can be incapacitating. For example, the troubling ocular symptoms can lead to the progressive inability to perform important daily activities, such as driving or working.
In an FDA news release, Wiley Chambers, MD, deputy director of the Division of Transplant and Ophthalmology Products in the FDA’s Center for Drug Evaluation and Research said, “[This] approval marks an important milestone for the treatment of thyroid eye disease. Currently, there are very limited treatment options for this potentially debilitating disease. This treatment has the potential to alter the course of the disease, potentially sparing patients from needing multiple invasive surgeries by providing an alternative, non surgical treatment option.”
Tepezza was approved based on the results of two studies consisting of a total of 170 patients with active thyroid eye disease who were randomized to either receive Tepezza or a placebo. Of the patients who were administered Tepezza, 71% in first study and 83% in the second study demonstrated a greater than two millimeter reduction in proptosis (eye protrusion) compared to only 20% and 10% of subjects who received placebo, respectively.
The most common adverse reactions observed in patients treated with Tepezza were muscle spasm, nausea, alopecia (hair loss), diarrhea, fatigue, hyperglycemia (high blood sugar), hearing loss, dry skin, dysgeusia (altered sense of taste) and headache. Tepezza should not be used if pregnant, and women of child-bearing potential should have their pregnancy status verified prior to beginning treatment and should be counseled on pregnancy prevention during treatment and for 6 months following the last dose of Tepezza.
In the video below, Gary Joseph Lelli, MD of the Department of Ophthalmology at Weill Cornell Medicine discusses the most common symptoms observed in thyroid eye disease, as well as the mostly likely clinicians that usually diagnose this rare condition.
