Perspective from Marius N. Stan, MD
January 21, 2020
3 min read

FDA approves teprotumumab for thyroid eye disease

Perspective from Marius N. Stan, MD
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Raymond Douglas

The FDA on Tuesday approved the human monoclonal antibody teprotumumab for the treatment of adults with thyroid eye disease, marking the first drug approved for the condition, according to an agency press release.

“This is a great announcement for all the patients and families affected by thyroid eye disease,” Raymond Douglas, MD, PhD, director of the orbital and thyroid eye disease program at Cedars-Sinai Medical Center, told Healio. “For the first time, there is an FDA-approved medication shown to reverse this terrible disease. We appreciate all the efforts of the patients, investigators and FDA for making this come to fruition.”
Thyroid eye disease, a condition most commonly associated with Graves’ hyperthyroidism, typically comes with a range of symptoms that can make everyday tasks challenging and quickly reduce quality of life. Within 18 months of a diagnosis of Graves’ disease, approximately 25% of patients will develop thyroid eye disease, with symptoms including proptosis, the main cause of morbidity, as well as eyelid retraction, strabismus and, occasionally, compressive optic neuropathy.

Teprotumumab (Tepezza, Horizon Therapeutics), an insulin-like growth factor-1 receptor inhibitor, is a fully human monoclonal antibody developed to address a substantial unmet need for patients with thyroid eye disease. The drug blocks the inflammatory/autoimmune pathophysiology that underlies thyroid eye disease.

As Healio previously reported, the Dermatologic and Ophthalmic Drugs Advisory Committee of the FDA voted 12-0 in December in favor of recommending approval of a biologics license application for teprotumumab. At the time, committee members before casting votes expressed concern about the small patient population studied — fewer than 90 adults treated with infusions of teprotumumab have been enrolled in controlled clinical trials — and several adverse events reported, among them hearing loss, muscle spasms, hyperglycemia and infections. In briefing materials, the FDA noted the population was considerably smaller than the common safety database of greater than 300 patients treated with a course of therapy. However, committee members ultimately agreed that the benefits of the drug outweighed any potential risks.

"Today's approval marks an important milestone for the treatment of thyroid eye disease,” Wiley Chambers, MD, deputy director of the division of transplant and ophthalmology products in the FDA's Center for Drug Evaluation and Research, said in the release. “Currently, there are very limited treatment options for this potentially debilitating disease. This treatment has the potential to alter the course of the disease, potentially sparing patients from needing multiple invasive surgeries by providing an alternative, nonsurgical treatment option.”


Among adults who received teptotumumab, 71% in Study 1 and 83% in Study 2 demonstrated a greater than 2 mm reduction in proptosis compared with 20% and 10% of participants who received placebo, respectively.

The most common adverse reactions observed among patients treated with teptotumumab are muscle spasm, nausea, alopecia, diarrhea, fatigue, hyperglycemia, hearing loss, dry skin, dysgeusia and headache.

In an interview during the AACE Annual Scientific and Clinical Congress in April, Douglas called teprotumumab a therapy that marks a “defining line” for how to move forward with the treatment of thyroid eye disease in the future. He added that the therapy could potentially replace surgery for many patients, including those with more advanced disease.

“Not all surgery for thyroid eye disease is created equal,” Douglas told Healio. “The more surgery you do, the more risk you take for causing double vision. These patients often have five to seven surgeries to improve this disease. If you could diminish that in a large number of these patients to zero or a couple, that would be revolutionary.”
The FDA granted this application priority review, in addition to fast track and breakthrough therapy designation. Additionally, teprotumumab received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases or conditions. Development of this product was also supported by the FDA Orphan Products Grants Program, which provides grants for clinical studies on safety and efficacy of products for use in rare diseases or conditions. – by Regina Schaffer

Disclosures: Douglas reports he serves as a consultant for Horizon Pharma.