Teprotumumab for the Treatment of Active Thyroid Eye Disease

Thyroid eye disease (TED) is a debilitating autoimmune condition with no FDA approved medical therapy. This randomized, double masked, placebo-controlled, phase 3 trial evaluated the efficacy and safety of teprotumumab, an insulin like growth factor I receptor (IGF-IR) inhibitor, in 83 patients with active TED. Participants received intravenous teprotumumab or placebo every 3 weeks for 21 weeks. At week 24, teprotumumab significantly improved proptosis (83% vs. 10% response rate), Clinical Activity Score (59% vs. 21% achieved minimal inflammation), diplopia (68% vs. 29% response), and quality of life (mean GO-QOL score change: 13.79 vs. 4.43 points) compared to placebo. Adverse events were mostly mild to moderate. The study demonstrates teprotumumab as a promising targeted therapy for active TED.

Thyroid eye disease (TED) is a debilitating autoimmune condition with no FDA approved medical therapy. This randomized, double masked, placebo-controlled, phase 3 trial evaluated the efficacy and safety of teprotumumab, an insulin like growth factor I receptor (IGF-IR) inhibitor, in 83 patients with active TED. Participants received intravenous teprotumumab or placebo every 3 weeks for 21 weeks. At week 24, teprotumumab significantly improved proptosis (83% vs. 10% response rate), Clinical Activity Score (59% vs. 21% achieved minimal inflammation), diplopia (68% vs. 29% response), and quality of life (mean GO-QOL score change: 13.79 vs. 4.43 points) compared to placebo. Adverse events were mostly mild to moderate. The study demonstrates teprotumumab as a promising targeted therapy for active TED.