First Treatment for Thyroid Eye Disease Approved by FDA

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The U.S. Food and Drug Administration has approved teprotumumab-trbw (Tepezza, Horizon Therapeutics) for the treatment of adults with thyroid eye disease, a rare condition where the muscles and fatty tissues behind the eye become inflamed, causing the eyes to be pushed forward and bulge outwards (proptosis). The drug approval, which was announced yesterday, represents the first drug approved for the treatment of thyroid eye disease. 

“Today’s 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,” said Wiley Chambers, M.D., deputy director of the division of transplant and ophthalmology products in the FDA’s Center for Drug Evaluation and Research. “Additionally, thyroid eye disease is a rare disease that impacts a small percentage of the population, and for a variety of reasons, treatments for rare diseases are often unavailable. This approval represents important progress in the approval of effective treatments for rare diseases, such as thyroid eye disease.”

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. In the treatment group, 71 percent in the first study and 83 percent in the second study demonstrated a greater than 2 millimeter reduction in proptosis (eye protrusion) as compared to 20 percent and 10 percent of subjects who received placebo, respectively.

The FDA granted this application Priority Review, in addition to Fast Track and Breakthrough Therapy Designation. Additionally, Tepezza received Orphan Drug designation.