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Spotlight on TEPEZZA®

Kerri Fitzgerald
Tepezza

This Spotlight Series article is editorially independent content.

Thyroid eye disease (TED) is a progressive and vision-threatening rare autoimmune disease most associated with Graves’ disease. Risk factors for more severe disease include older age (>65 years), tobacco use, and a high level of thyroid autoantibodies. Signs and symptoms include orbital/periocular pain, swelling, and redness with proptosis and diplopia seen in more severe disease. Increasing levels of proptosis and diplopia are associated with worsening quality of life.1

TEPEZZA®: Targets Cause of TED

Advances in the understanding of the pathophysiology of TED have shifted the focus from supportive therapy to disease-modifying treatment. In 2020, the US Food and Drug Administration (FDA) approved the first treatment for TED. TEPEZZA (teprotumumab-trbw; Amgen) is an insulin-like growth factor 1 receptor indicated for the treatment of TED regardless of disease activity or duration.2,3

Efficacy Data

TEPEZZA has demonstrated efficacy in treating patients with TED across a broad range of clinical manifestations.4,5

Studies 1 and 2

The FDA approval was based on the results of two 24-week, randomized, double-masked, placebo-controlled, phase 2 and 3 studies (Study 1 and 2). A total of 170 patients with active TED were randomized to TEPEZZA or placebo. At week 24, more patients receiving TEPEZZA in Study 1 and 2 (71% and 83%, respectively) demonstrated ≥2-mm reduction in proptosis compared with placebo (20% and 10%, respectively).2,5

In Study 2, mean change in proptosis from baseline was observed at as early as 6 weeks and showed continued improvement through week 24 (see FIGURE 1). In both studies, TEPEZZA also completely resolved diplopia in twice as many patients compared with placebo (53% vs 25%).2

Phase 4 Study

A randomized, double-masked, placebo-controlled, phase 4 study of 62 patients with low disease activity and long-duration TED showed significant and continuous reduction in proptosis with TEPEZZA (see FIGURE 2). In the intent-to-treat analysis, most patients receiving TEPEZZA achieved ≥2-mm reduction in proptosis at week 24 versus placebo (62% vs 25%, respectively).6

OPTIC-X

TEPEZZA continued to show improvement over time in the OPITC-X extension study. The following patients were included: patients taking TEPEZZA who were proptosis nonresponders at OPTIC week 24 or proptosis responders at week 24 but flared during 48-week follow-up period. A flare was defined as patients who lost at ≥2 mm of their week 24 proptosis improvement during the 48-week off-treatment follow-up period, even if their proptosis was still substantially better than at baseline of OPTIC, or who had a significant increase in the number of inflammatory signs or symptoms without worsening proptosis.7

For patients who had proptosis response at week 24 and then experienced a flare, 63% showed a ≥2-mm reduction from baseline with a second course of TEPEZZA at week 24.7

Safety and Tolerability

TEPEZZA has a proven efficacy with a demonstrated safety and tolerability profile.2,5,8 Most adverse events (AEs) were mild or moderate, manageable, and resolved during or after treatment.5,8 The most common adverse reactions reported in ≥5% of patients were:2

  • Muscle spasms
  • Nausea
  • Alopecia
  • Diarrhea
  • Fatigue
  • Hyperglycemia
  • Hearing impairment
  • Dry skin
  • Dysgeusia
  • Headache
  • Decreased weight
  • Nail disorder
  • Menstrual disorders

Additionally, phase 4 data support the safety and tolerability in Study 1 and 2. Overall, 98% of patients treated with TEPEZZA did not discontinue treatment due to AEs at week 24, and no new safety signals were identified.6

Dosing and Administration

The recommended dose of TEPEZZA is an intravenous (IV) infusion of 10 mg/kg for the initial dose followed by an IV infusion of 20 mg/kg every 3 weeks for 7 additional infusions. Because each patient responds differently to treatment, patients should complete the full TEPEZZA treatment course of 8 IV infusions as studied in clinical trials. TEPEZZA may be administered at an infusion center, ophthalmologist’s office, hospital, or the patient’s home.2

For more information, visit www.tepezzahcp.com.

References

  1. Smith TJ, Cockerham K, Lelli G, et al. Utility assessment of moderate to severe thyroid eye disease health states. JAMA Ophthalmol. 2023;141(2):159-166. doi:10.1001/jamaophthalmol.2022.3225
  2. Package insert. Amgen Inc; 2025.
  3. FDA approves first treatment for thyroid eye disease. US Food & Drug Administration. January 21, 2020. Accessed April 1, 2025. https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-thyroid-eye-disease
  4. Ugradar S, Kang J, Kossler AL, et al. Teprotumumab for the treatment of chronic thyroid eye disease. Eye (Lond). 2022;36(8):1553-1559. doi:10.1038/s41433-021-01593-z
  5. Douglas RS, Kahaly GJ, Patel A, et al. Teprotumumab for the treatment of active thyroid eye disease. N Engl J Med. 2020;382(4):341-352. doi:10.1056/NEJMoa1910434
  6. Douglas RS, Couch S, Wester ST, et al. A randomized, quadruple-masked, placebo-controlled, multicenter trial to evaluate the efficacy and safety of teprotumumab in patients with chronic (inactive/low CAS) thyroid eye disease. J Endocr Soc. 2023;7(suppl 1):A1026-A1027.
  7. Douglas RS, Kahaly GJ, Ugradar S, et al. Teprotumumab efficacy, safety, and durability in longer-duration thyroid eye disease and re-treatment: OPTIC-X study. 2022;129(4):438-449. doi:10.1016/j.ophtha.2021.10.017
  8. Smith TJ, Kahaly GJ, Ezra DG, et al. Teprotumumab for thyroid-associated ophthalmopathy. N Engl J Med. 2017;376(18):1748-1761. doi:10.1056/NEJMoa1614949

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