Teprotumumab for thyroid eye diseaseMarch 6, 2020
The US FDA approved teprotumumab-trbw for the treatment of adults with thyroid eye disease. The approval represents the first drug approved for the treatment of this rare condition, Horizon Therapeutics said.
Teprotumumab was approved based on the results of two studies
(Study 1 and 2) consisting of a total of 170 patients with active thyroid eye disease who were randomized to either receive teprotumumab or a placebo. Of the patients who were administered teprotumumab, 71% in Study 1 and 83% in Study 2 demonstrated a greater than 2 millimeter reduction in proptosis 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. Additionally, teprotumumab received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases or conditions.