Teprotumumab Treatment for Thyroid-Associated Ophthalmopathy

Thyroid-associated ophthalmopathy (TAO), an autoimmune process affecting the tissues surrounding the eye, most commonly develops in individuals with Graves' disease. It is disfiguring, can cause vision loss, and dramatically lessens the quality of life in patients. There has been an absence of approved medical therapies for TAO with proven effectiveness and safety in multicenter, placebo-controlled, and adequately powered clinical trials.

The following is a brief overview of the rationale for developing a monoclonal antibody inhibitor of the insulin-like growth factor-I receptor into a treatment for TAO. This area of fundamental research has yielded an effective and safe medication, namely teprotumumab, based on two multicenter, placebo-controlled trials. Teprotumumab, marketed as Tepezza, has been approved recently by the US Food and Drug Administration for the treatment of TAO. Given its remarkable effectiveness, Tepezza is poised to become the first-line standard of care for TAO.

Introduction of Tepezza into our armamentarium of therapeutic strategies for TAO represents a paradigm shift in the management of the disease. I proffer that the drug will replace glucocorticoids as a first-line treatment for TAO.