Safety profile of teprotumumab-trbw, the first and only FDA-approved treatment for thyroid-associated ophthalmopathy

Teprotumumab (Tepezza), a monoclonal antibody targeting the insulin-like growth factor 1 receptor (IGF-IR), was approved in January 2020 by the US Food and Drug Administration for treatment of thyroid-associated ophthalmopathy (TAO). The drug was found to be effective and generally well-tolerated in two registered clinical trials and subsequent post-marketing trials.

A search of PubMed and other databases of medical and scientific literature was conducted. Key search terms included 'teprotumumab,' 'Tepezza,' 'thyroid-associated ophthalmopathy treatment,' and 'Graves' disease treatment.' Two adverse events (AEs), auditory abnormalities and hyperglycemia, were the focus of this review. As of 7 December 2025, 392 citations were identified in PubMed using teprotumumab as the search term. Of these, 29 publications primarily reported AEs or safety concerns associated with teprotumumab. The two most consequential AEs, hearing dysfunction and hyperglycemia, were identified in phase 2 and phase 3 clinical trials.

Teprotumumab, the only FDA-approved therapy for TAO, is generally effective. It has been associated with several side effects; the most concerning are hearing dysfunction and hyperglycemia. Due diligence in pretreatment, intra-treatment and post-treatment monitoring is essential. Detailed benefit-risk analysis should be shared with all patients for whom use of the drug is being considered.