Thyroid-associated ophthalmopathy: emergence of teprotumumab as a promising medical therapy

Publication date: Available online 31 January 2020Source: Best Practice & Research Clinical Endocrinology & MetabolismAuthor(s): Terry J. SmithAbstractThyroid-associated ophthalmopathy (TAO) remains a vexing autoimmune component of Graves’ disease that can diminish the quality of life as a consequence of its impact on visual function, physical appearance and emotional well-being. Because of its relative rarity and variable presentation, the development of highly effective and well-tolerated medical therapies for TAO has been slow relative to other autoimmune diseases. Contributing to the barriers of greater insight into TAO has been the historical absence of high-fidelity preclinical animal models. Despite these challenges, several agents, most developed for other diseases, have found their way into consideration for use in active TAO through repurposing from their original purposes. Among these, teprotumumab is a fully human monoclonal antibody that acts as a β-arrestin agonist of the insulin-like growth factor I receptor. It has shown remarkable effectiveness in moderate to severe, active TAO in two completed multicenter, double masked, and placebo controlled clinical trials. The drug exhibits a favorable safety profile. Should teprotumumab become approved by the U.S. F.D.A, it may rapidly become the first line therapy for this disfiguring and potentially blinding condition.

Source: Best Practice and Research Clinical Endocrinology and Metabolism - January 31, 2020 Category: Endocrinology Source Type: research