Teprotumumab
| Monoclonal antibody | |
|---|---|
| Type | Whole antibody |
| Source | Human |
| Target | IGF-1R |
| Clinical data | |
| Trade names | Tepezza |
| Other names | Teprotumumab-trbw, RG-1507 |
| AHFS/Drugs.com | Monograph |
| MedlinePlus | a620025 |
| License data |
|
| Routes of administration | Intravenous |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| DrugBank | |
| ChemSpider |
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| UNII | |
| KEGG | |
| ChEMBL | |
| ECHA InfoCard | 100.081.384 |
| Chemical and physical data | |
| Formula | C6476H10012N1748O2000S40 |
| Molar mass | 145639.97 g·mol−1 |
  (what is this?) (verify) | |
Teprotumumab, sold under the brand name Tepezza, is a medication used to treat thyroid eye disease (Graves' eye disease), a rare condition where the muscles and fatty tissues behind the eye become inflamed, causing the eyes to bulge outwards.[3][4] It is a human monoclonal antibody developed by Genmab and Roche[5] for tumor treatment but was later developed by River Vision Development Corporation and Horizon Therapeutics to be used for ophthalmic uses.[6] It binds to IGF-1R.[2]
The most common side effects are muscle spasm, nausea, hair loss, diarrhea, fatigue, high blood sugar, hearing loss, dry skin, altered sense of taste, and headache.[4] Teprotumumab should not be used if pregnant.[4]
Teprotumumab was approved for medical use in the United States in January 2020.[4][7][8][9] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.[10]
Medical use
[edit]Teprotumumab is indicated for the treatment of thyroid eye disease.[2]
History
[edit]In a multi-center, randomized trial in participants with active Graves' ophthalmopathy, teprotumumab was more effective than placebo.[11] In February 2019, Horizon announced results from a Phase III confirmatory trial evaluating teprotumumab for the treatment of active thyroid eye disease (TED). The study met its primary endpoint, showing more participants treated with teprotumumab compared with placebo had a meaningful improvement in proptosis, or bulging of the eye: 82.9% of teprotumumab participants compared to 9.5% of placebo participants achieved the primary endpoint of a 2 mm or more reduction in proptosis (p<0.001). Proptosis is the main cause of morbidity in TED. All secondary endpoints were also met and the safety profile was consistent with the Phase II study of teprotumumab in TED.[12] In July 2019, Horizon submitted a biologics license application to the US Food and Drug Administration (FDA) for teprotumumab for the treatment of active thyroid eye disease.[13]
Teprotumumab was first investigated for the treatment of solid and hematologic tumors, including breast cancer, Hodgkin's and non-Hodgkin's lymphoma, non-small cell lung cancer, and sarcoma.[14]
Teprotumumab-trbw was approved based on the results of two clinical trials (Trial 1/ NCT01868997 and Trial 2/ NCT03298867) of 170 subjects with active TED who were randomized to either receive teprotumumab-trbw or a placebo.[4][8] Of the subjects who were administered Tepezza, 71% in study 1 and 83% in study 2 demonstrated a greater than 2 mm reduction in proptosis (eye protrusion) as compared to 20% and 10% of subjects who received placebo, respectively.[4] The trials were conducted at 28 sites in Europe and United States.[8] Teprotumumab-trbw was approved for use in the United States in January 2020, for the treatment of adults with thyroid eye disease.[4][9] The FDA granted the application for teprotumumab-trbw fast-track, breakthrough therapy, priority review, and orphan drug designations.[4] The FDA granted the approval of Tepezza to Horizon Therapeutics Ireland DAC.[4]
Society and culture
[edit]Legal status
[edit]In April 2025, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Tepezza, intended for the treatment of adults with moderate to severe thyroid eye disease.[15] The applicant for this medicinal product is Amgen Europe B.V.[15]
The CHMP's opinion is based on data from three randomized, placebo-controlled trials in a total of 225 participants with active thyroid eye disease, and one trial in 62 participants with chronic thyroid eye disease.[16] After 24 weeks, participants treated with teprotumumab experienced a significant reduction (-2 to -2.3 mm) in protrusion of the eyeball from the eye socket (proptosis) and in the clinical activity score, a standard tool to evaluate inflammatory signs and symptoms of thyroid eye disease, compared to participants treated with placebo.[16] The reduction in proptosis was smaller (-1.5mm) in participants with chronic thyroid eye disease.[16]
References
[edit]- ^ "Tepezza (Amgen Australia Pty Ltd)". Therapeutic Goods Administration (TGA). 16 April 2025. Retrieved 3 May 2025.
- ^ a b c "Tepezza- teprotumumab injection, powder, lyophilized, for solution". DailyMed. 24 July 2023. Retrieved 20 December 2023.
- ^ Shah K, Charitou M (1 July 2022). "A Novel Case of Hyperglycemic Hyperosmolar State After the Use of Teprotumumab in a Patient With Thyroid Eye Disease". AACE Clinical Case Reports. 8 (4): 148–149. doi:10.1016/j.aace.2022.01.004. PMC 9363510. PMID 35959086.
- ^ a b c d e f g h i "FDA approves first treatment for thyroid eye disease" (Press release). U.S. Food and Drug Administration (FDA). 21 January 2020. Retrieved 21 January 2020.
This article incorporates text from this source, which is in the public domain.
- ^ Ali F, Chorsiya A, Anjum V, Ali A (April 2021). "Teprotumumab (Tepezza): from the discovery and development of medicines to USFDA approval for active thyroid eye disease (TED) treatment". International Ophthalmology. 41 (4): 1549–1561. doi:10.1007/s10792-021-01706-3. PMID 33481154. S2CID 231670915.
- ^ "Horizon Pharma plc Announces Agreement to Acquire River Vision Development Corp. and Teprotumumab (RV001), a Biologic in Late-Stage Development for Rare Eye Disease" (Press release). Horizon Pharma. 8 May 2017. Retrieved 22 August 2022 – via GlobeNewswire.
- ^ "Teprotumumab-trbw: FDA-Approved Drugs". U.S. Food and Drug Administration (FDA). Retrieved 28 January 2020.
- ^ a b c "Drug Trial Snapshot: Tepezza". U.S. Food and Drug Administration (FDA). 29 January 2020. Retrieved 29 January 2020. This article incorporates text from this source, which is in the public domain.
- ^ a b "Drug Approval Package: Tepezza". U.S. Food and Drug Administration (FDA). 4 June 2021. Retrieved 20 December 2023.
- ^ "New Drug Therapy Approvals 2020". U.S. Food and Drug Administration (FDA). 31 December 2020. Retrieved 17 January 2021. This article incorporates text from this source, which is in the public domain.
- ^ Smith TJ, Kahaly GJ, Ezra DG, Fleming JC, Dailey RA, Tang RA, et al. (May 2017). "Teprotumumab for Thyroid-Associated Ophthalmopathy". The New England Journal of Medicine. 376 (18): 1748–1761. doi:10.1056/NEJMoa1614949. PMC 5718164. PMID 28467880.
- ^ "Horizon Pharma plc Announces Phase 3 Confirmatory Trial Evaluating Teprotumumab (OPTIC) for the Treatment of Active Thyroid Eye Disease (TED) Met Primary and All Secondary Endpoints". Horizon Pharma (Press release). Retrieved 22 March 2019.
- ^ "Horizon Therapeutics plc Submits Teprotumumab Biologics License Application (BLA) for the Treatment of Active Thyroid Eye Disease (TED)". Horizon Therapeutics (Press release). Retrieved 27 August 2019.
- ^ Clinical trial number NCT01868997 for "Teprotumumab (RV 001) Treatment in Patients With Active Thyroid Eye Disease" at ClinicalTrials.gov
- ^ a b "Tepezza EPAR". European Medicines Agency (EMA). 25 April 2025. Retrieved 2 May 2025. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ^ a b c "First treatment against severe thyroid eye disease" (Press release). European Medicines Agency (EMA). 25 April 2025. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
