FDA Approves Update to Indication Language for Teprotumumab in Thyroid Eye Disease

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According to Horizon Therapeutics, the updated indication language reinforces the importance of access for patients across the full spectrum of TED activity or duration.

Today, Horizon Therapeutics announced approval of an update to the indication language for teprotumumab-trbw (TEPEZZA®) to specify its use in patients with thyroid eye disease (TED) regardless of disease activity or duration.1

The therapy is the first and only medicine approved by the US Food and Drug Administration (FDA) for the treatment of TED. Now, this updated language serves as reinforcement to the importance of unrestricted access for patients across the full spectrum of TED, according to the company’s release.

“We worked closely with the FDA on this important label update, which further reinforces the potential benefit of TEPEZZA in people impacted by Thyroid Eye Disease regardless of disease activity or duration,” said Tim Walbert, chairman, president and chief executive officer, Horizon.1 “While TEPEZZA already had a broad indication for the treatment of TED, the new indication language now references treatment regardless of disease activity or duration. This creates an opportunity to ease the access burden for patients and physicians with the goal of decreasing time to therapy for patients who may benefit from TEPEZZA.”

This update follows the release of positive topline results from a randomized, double-masked, placebo-controlled phase 4 clinical trial. the findings suggested that patients with an initial diagnosis of TED between 2-10 years (mean duration, 5.2 years) and with low clinical activity score (CAS; mean, 0.4) achieved a statistically significant reduction in proptosis from baseline at Week 24 after receiving teprotumumab compared to placebo.

As previously reported, data showed teprotumumab treatment was associated with a 2.41 mm reduction in proptosis from baseline compared with 0.92 mm for those receiving placebo (P = .0004). Additionally, 62% of teprotumumab-treated patients had a clinically meaningful improvement at Week 24 (≥2 mm) compared with 25% of patients receiving placebo (P = .0134).

There were no new safety signals observed in the phase 4 trial, with the therapy linked to infusion reactions in approximately 4% of patients and increased blood glucose or hyperglycemia in approximately 10% of patients.

In a statement, Raymond Douglas, MD, PhD, principal investigator, and director of the Orbital and Thyroid Eye Disease Program, Cedars-Sinai Medical Center, noted that the new positive phase 4 clinical evidence suggests the importance of physicians assessing all patients with TED to determine if teprotumumab treatment may be an option.2

“It is important to specifically ask patients if their symptoms are interfering with their ability to work, socialize, and go about daily activities,” Douglas said.2 “These conversations can help physicians uncover the true burden of the disease and need for treatment, regardless of how much inflammation they have behind the eye or how long they have been living with the disease.”

The company plans to present the data at an upcoming medical congress.


  1. Horizon Therapeutics plc announces FDA approval of an update to the indication language for TEPEZZA® (teprotumumab-TRBW) to specify its use in thyroid eye disease (TED) patients regardless of disease activity or duration. Business Wire. (2023, April 14). Retrieved April 14, 2023, from
  2. 2. Horizon Therapeutics plc announces positive topline data from Tepezza® (teprotumumab-TRBW) phase 4 clinical trial in patients with chronic/low clinical activity score (CAS) Thyroid Eye Disease (ted). Horizon Therapeutics plc. (2023, April 10). Retrieved April 10, 2023, from