Data From Phase 3 Clinical Trial of Teprotumumab Demonstrates Improvements in Proptosis, Quality of Life

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Patients with active thyroid eye disease who were treated with teprotumumab had significant improvements in proptosis, diplopia, and quality of life compared to placebo, according to an article published in the New England Journal of Medicine. Teprotumumab, which received FDA approval on January 21, 2020, is the first approved treatment for adults with thyroid eye disease.

In the phase 3 OPTIC clinical trial evaluating the insulin-like growth factor I receptor (IGF-IR) teprotumumab-trbw for thyroid eye disease, patients were assigned to receive intravenous infusions of teprotumumab (10 mg per kilogram of body weight for the first infusion and 20 mg per kilogram for subsequent infusions) (n = 41) or placebo (n = 42) once every 3 weeks for 21 weeks. At 24 weeks the last trial visit for analysis occurred.

At 24 weeks, 83% of patients treated with teprotumumab had a ≥ 2 mm reduction of proptosis versus 10% in the group treated with placebo (P < 0.001). Overall response (78% vs 7%), Clinical Activity Score of 0 or 1 (59% vs 21%), mean change in proptosis (−2.82 mm vs −0.54 mm), diplopia response (68% vs 29%), and mean change in Graves’ ophthalmopathy-specific quality-of-life overall score (13.79 points vs 4.43 points) (P ≤0.001 for all), were significantly better with teprotumumab than with placebo. In 6 patients who received teprotumumab who underwent orbital imaging, reductions in extraocular muscle, orbital fat volume, or both were observed.

“Thyroid Eye Disease is a rare, devastating autoimmune disease that is not adequately treated, leaving patients to struggle for years until they become candidates for surgeries that are not only complex, but often don’t fully restore vision or appearance,” said Raymond Douglas, MD, PhD, director of the Orbital and Thyroid Eye Disease Program, Cedars-Sinai Medical Center and co-principal investigator of the OPTIC trial in a press release. “In this clinical trial, we saw statistically significant improvements across critical symptoms – including proptosis and diplopia – at the first patient assessment at six weeks of treatment, and those improvements continued over the 24-week treatment period. TEPEZZA, which was just recently approved by the FDA, has the potential to significantly change the treatment paradigm in a disease where patients have historically had to watch and wait in pain as symptoms progress and put them at risk for serious vision impairment.”



Douglas, Kahaly GJ, Patel A, et al. Teprotumumab for the treatment of active thyroid eye disease. N Engl J Med. 2020;382(4):341-352.

Horizon Therapeutics. The New England Journal of Medicine publishes comprehensive data from phase 3 clinical trial (OPTIC) of TEPEZZA™ (teprotumumab-trbw) for thyroid eye disease. 2020. Available at:  https://www.businesswire.com/news/home/20200122005850/en/New-England-Journal-Medicine-Publishes-Comprehensive-Data





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