Teprotumumab gets FDA go-ahead for thyroid eye disease



The Food and Drug Administration has approved teprotumumab-trbw (Tepezza) as the first drug for treating thyroid eye disease, according to a press release.

A stamp saying "FDA approved." Olivier Le Moal/Getty Images

Thyroid eye disease is a rare, progressive, autoimmune condition that causes the eyes to bulge (proptosis) and can lead to blindness. Until now, treatment has focused on managing its symptoms – which can include eye pain, double vision, or sensitivity to light – with steroids, and in some cases, multiple invasive surgeries.

The human monoclonal antibody and a targeted inhibitor of the insulinlike growth factor-1 receptor is administered to patients once every 3 weeks, for a total of eight infusions, according to a statement from Horizon Therapeutics, which manufactures the drug.

The approval was based on the findings from two similarly designed, parallel-group studies (Studies 1 and 2) involving 170 patients with thyroid eye disease who were randomized to receive either teprotumumab or placebo. Of those receiving the study drug, 71% in Study 1 and 83% in Study 2 had a reduction of more than 2 mm in eye protrusion, compared with 20% and 10%, respectively, among the placebo participants.

The most common adverse reactions in patients receiving teprotumumab were muscle spasm, nausea, alopecia, diarrhea, fatigue, and hyperglycemia. The treatment is contraindicated for pregnancy.

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