By Serena Gordon
WEDNESDAY, January 22, 2020 (HealthDay News) – The recently approved teprotumumab drug may offer hope to adults with thyroid eye disease, a rare and potentially blinding condition.
It is the first treatment specifically approved for thyroid eye disease. The drug was approved Tuesday by the U.S. Food and Drug Administration. UU. It will be marketed under the Tepezza brand.
"This may be a drug that offers a new approach and a paradigm shift," said Dr. Raymond Douglas, lead author of a study that 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 will not have to suffer as they have in the past. With treatment, they will not be blind. They will not be disfigured. They can even watch their children play soccer when it is windy. "Douglas explained. (Without treatment, the wind may be drying your eyes too much.)
In people with the condition, inflammation in the back of the eye causes the eyes to bulge. The swelling can also compress the optic nerve, causing vision problems, said Douglas, a professor of surgery in the ophthalmology division at Cedars-Sinai Medical Center in Los Angeles.
Thyroid eye disease usually develops in people with an overactive thyroid gland caused by Graves' disease. The disorder can cause redness, swelling and a sandy feeling in the eyes, says the American Thyroid Association. It can also cause dry eyes or excessive tearing, double vision and sensitivity to light. One of the most notable symptoms is the bulging of the eyes, and it can be difficult to completely close the eyes.
Because thyroid eye disease is rare, the FDA granted this drug the status of "orphan drug." This means that companies receive incentives to encourage the development of drugs for rare diseases. The drug also received FDA priority review, fast-track designations and innovative therapy to help the manufacturer bring the medication to market.
Drug approval was based on the results of two studies that included 170 people with active thyroid disease. The most recent study was a phase 3 clinical trial published on January 22 in the New England Journal of Medicine and directed by Douglas. This study included more than 80 people with active thyroid disease who were randomly assigned to receive teprotumumab or a placebo.
The drug was administered intravenously once a week for three weeks over a period of 21 weeks. Douglas said intravenous infusions take about an hour.
Douglas said teprotumumab started working after only two doses. After 24 weeks, 83% of people who took the medication had an appreciable reduction in eye protuberance, more than 2 millimeters, compared to only 10% of those who received a placebo.
The overall response rate was 78% among those who took the medication compared to 7% of people who took a placebo. The quality of life was much higher for people who took the medication, the study found.
Douglas said treatment with the drug reversed double vision as well as surgery.
Side effects were minimal and well tolerated, according to Douglas. Some of the most common side effects include muscle cramps, nausea, hair loss and fatigue. The medicine should not be used in pregnancy.
Dr. Harsha Reddy, an ophthalmic plastic surgeon at the eye and ear nurse in New York at Mount Sinai, was not involved in the current study, but is familiar with the findings.
"I think this will be a revolution in the treatment of thyroid eye disease," he said.
"Until now, [doctors] He could only wait until the thyroid disease had affected his patients, and both the patients and the doctors were frustrated by the lack of treatment options during that waiting period, "Reddy explained.
"Now, for the first time, doctors will have the option to treat thyroid eye disease in its initial phase and prevent many of the devastating eye changes from occurring," Reddy added.