The newly approved drug teprotumumab can offer hope to adults with thyroid eye disease, a rare and potentially blinding condition.
It’s the first treatment specifically approved for thyroid eye disease. The drug was approved Tuesday, January 11, 2020 by the U.S. Food and Drug Administration. It will be marketed under the brand name Tepezza.
“This may be a drug that offers a new approach and a paradigm shift,” said Dr. Raymond Douglas, lead author of a study the FDA considered when making its final decision. “Currently, we use supportive therapy for people with thyroid eye disease, but their quality of life is diminished by their symptoms.
“Teprotumumab was extraordinarily effective. We hope that people with thyroid eye disease won’t have to suffer as they have in the past. With treatment, they’re not going to be blind. They’re not going to be disfigured. They can even watch their kids playing soccer when it’s windy,” Douglas explained. Without treatment, wind could be too drying to the eyes.
Thyroid eye disease causes inflammation in the back of the eye causing the eyes to bulge. The swelling may also squeeze the optic nerve, causing vision problems, said Douglas, a professor of surgery in the division of ophthalmology at Cedars-Sinai Medical Center in Los Angeles.
Thyroid eye disease typically develops in people with an overactive thyroid gland caused by Graves’ disease. Redness, swelling and a gritty feeling in the eyes are also classic symptoms, the American Thyroid Association says. Other conditions that can occur include dry eyes or excessive tearing, double vision and sensitivity to light. The most noticeable symptom, however, is the bulging of the eyes, and it may be difficult to completely close the eyes.
Because thyroid eye disease is rare, the FDA gave this drug “orphan drug” status. This means companies are provided incentives to encourage the development of drugs for rare diseases. The medication was also given the FDA’s priority review, fast track and breakthrough therapy designations to assist the manufacturer in bringing the drug to market.
The drug’s approval was based on results of two studies that included 170 people with active thyroid disease. The drug was administered intravenously once every week for three weeks over a 21-week period. Douglas said the IV infusions take about an hour.
Teprotumumab started working after just two doses, Douglas said. After 24 weeks, 83 percent of people on the drug had a measurable reduction in eye bulge — more than 2 millimeters — versus just 10 percent of those on a placebo.
The overall response rate was 78 percent among those taking the drug compared to 7 percent of people taking a placebo. Quality of life was much higher for people taking the drug, the study found.
Douglas said treatment with the drug reversed double vision as well as surgery does.
Side effects were minimal and well-tolerated, according to Douglas. Some of the more common side effects included muscle cramps, nausea, hair loss and fatigue. The drug should not be used in pregnancy.
Learn more about thyroid eye disease from the American Thyroid Association.
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