Published in Research

Real-world experience shows Tepezza (teprotumumab-trbw) was effective in treating people with DON

Horizon Therapeutics plc announced results of an analysis of real-world treatment with TEPEZZA in Thyroid Eye Disease (TED) patients with DON, the majority of whom had not previously responded to other treatments. DON is a complication of TED characterized by thyroid-related impairment of visual function and can lead to permanent sight loss related to optic nerve compression.1

TEPEZZA is the first and only medicine approved by the U.S. Food and Drug Administration (FDA) for the treatment of TED – a serious, progressive and potentially vision-threatening rare autoimmune disease.2,3 This analysis summarizes real-world experience of people living with TED and DON who were treated with TEPEZZA between January 2020 and September 2022.

During this period, 24 patients were identified from literature and the author’s clinical experience with most (22 out of 24) receiving treatment with oral or intravenous steroids, surgery and/or radiation prior to TEPEZZA.

After TEPEZZA treatment, meaningful improvement was observed in visual acuity in eyes with DON (mean lines of improvement of 3.7 – 23 patients, range 1-15, mean improvement in the mean deviation on visual field testing of -5.87 – 7 patients); proptosis (mean reduction of 4.4 mm – 19 patients); diplopia (mean improvement of 0.75 grade – 8 patients); and Clinical Activity Score (CAS) (mean reduction of 5.1 – 17 patients).

Reported adverse events were consistent with TEPEZZA clinical trials and included fatigue, dysgeusia, hearing issues, nausea, hyperglycemia and muscle spasms.

“We know how devastating [TED] can be for patients, and the risk of blindness in those with dysthyroid optic neuropathy is one of the most worrisome complications, especially when traditional therapies fail,” said Madhura A. Tamhankar, MD, study author and associate professor of ophthalmology and neurology at the Hospital of the University of Pennsylvania. “These data are encouraging for physicians looking for an option to treat patients at the highest risk of losing their sight and provide evidence that TEPEZZA can help improve symptoms and preserve visual acuity.”

A recent health state analysis in JAMA Ophthalmology assessed the burden of TED, revealing people with moderate-to-severe disease experience substantial disutility in their day-to-day lives.

In the study of over 100 general population participants, the worst impact on quality of life was associated with the most severe disease state (constant diplopia and large proptosis).4

“The spectrum and severity of [TED] symptoms can vary greatly for people and often require significant intervention to avoid long-term repercussions and negative impact to everyday activity, especially when dysthyroid optic neuropathy is involved,” said Mandeep Kaur, MD, MS, senior vice president, global medical affairs and outcomes research, Horizon. “This research underscores our ongoing commitment to ensuring patients receive appropriate care that can result in improved clinical outcomes.”


1. Agarwal A, et al. Dysthyroid Optic Neuropathy. StatPearls. 2022.

2. TEPEZZA (teprotumumab-trbw) [prescribing information] Horizon. Barrio-Barrio J, et al. Graves' Ophthalmopathy: VISA versus EUGOGO Classification, Assessment, and Management. Journal of Ophthalmopathy. 2015;2015:249125.

3. Smith TJ, Cockerham K, Lelli G, et al. Utility Assessment of Moderate to Severe Thyroid Eye Disease Health States. JAMA Ophthalmol. Published online December 29, 2022. doi:10.1001/jamaophthalmol.2022.3225

4. Weightman DR, et al. Autoantibodies to IGF-1 Binding Sites in Thyroid Associated Ophthalmopathy. Autoimmunity. 1993;16(4):251–257. 5. Pritchard J, et al. Immunoglobulin Activation of T Cell Chemoattractant Expression in Fibroblasts from Patients with Graves’ Disease Is Mediated Through the Insulin-Like Growth Factor 1 Receptor Pathway. J Immunol. 2003;170:6348-6354.

6. Bartalena L, Kahaly GJ, Baldeschi L, et al. The 2021 European Group on Graves’ Orbitopathy (EUGOGO) Clinical Practice Guidelines for the Medical Management of Graves’ Orbitopathy [published online ahead of print]. Eur J Endocrinol. 2021 Jul 1:EJE-21-0479.R1. doi: 10.1530/EJE-21-0479.

7. McKeag D, et al. Clinical features of dysthyroid optic neuropathy: a European Group on Graves' Orbitopathy (EUGOGO) survey. Br J Ophthalmol. 2007;91:455-458.