The U.S. Food and Drug Administration approved Horizon Therapeutics's Tepezza (teprotumumab-trbw) 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 approval represents the first drug approved for the treatment of thyroid eye disease.
There are very limited treatment options for this potentially debilitating disease and this new treatment potentially spares patients from needing multiple invasive surgeries by providing an alternative, non surgical treatment option. Thyroid eye disease is a rare disease that impacts a small percentage of the population, making treatments often unavailable.
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 or difficulty closing the eye.
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 Study 1 and 83% in Study 2 demonstrated a greater than 2 millimeter reduction in eye protrusion as compared to 20% and 10% of subjects who received placebo, respectively.
The FDA granted this application priority review, in addition to fast track and breakthrough therapy designation. Tepezza also received orphan drug designation.
Read the FDA release