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Review
. 2021 Nov 25:17:1219-1230.
doi: 10.2147/TCRM.S303057. eCollection 2021.

Efficacy and Safety of Teprotumumab in Thyroid Eye Disease

Honeylen Maryl Teo  1 , Terry J Smith  2   3 , Shannon S Joseph  2
Affiliations
Review

Efficacy and Safety of Teprotumumab in Thyroid Eye Disease

Honeylen Maryl Teo et al. Ther Clin Risk Manag. .

Abstract

Thyroid eye disease (TED; also known as thyroid-associated ophthalmopathy) is an autoimmune condition with disabling and disfiguring consequences. Teprotumumab is the first and only medication approved by the United States Food and Drug Administration for the treatment of TED. We review the efficacy and safety of teprotumumab in TED, highlighting results from the 2 randomized, double-masked, placebo-controlled trials. Post-approval case reports of teprotumumab use in patients with compressive optic neuropathy (CON) and inactive TED were similarly favorable to those from the trials. The preliminarily results of teprotumumab for CON and inactive TED should be investigated in formal clinical trials. Teprotumumab should be avoided in pregnancy. Evidence also suggests that teprotumumab may exacerbate pre-existing inflammatory bowel disease, worsen hyperglycemia, and be associated with hearing impairment. Patients at risk for these adverse events need to be closely monitored with baseline and periodic assessments.

Keywords: Graves’ ophthalmopathy; anti-IGF-IR; autoimmune; monoclonal antibody; thyroid-associated ophthalmopathy.

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Conflict of interest statement

Dr. Terry J. Smith has been issued the following US patents, held by University of California Los Angeles and Los Angeles Biomedical Institute and covering the use of insulin-like growth factor I receptor inhibition and teprotumumab in Graves’ disease and other autoimmune diseases: 6936426, 7998681, 8153121, and 8178304. He receives consulting fees from Horizon Therapeutics and Immunovant outside the submitted work. His institution received research support from Horizon Therapeutics for participating in the clinical trials of teprotumumab for TED. Dr. Shannon S. Joseph receives consulting fees from Horizon Therapeutics outside the submitted work. The authors report no other conflicts of interest in this work.

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