Study Finds Treatment for Rare Eye Disease May Prevent Blindness

Patients with thyroid eye disease who used teprotumumab, an insulin-like growth factor I blocking antibody, experienced improvement in their symptoms, appearance, and quality of life, according to a study recently published in The New England Journal of Medicine.

Thyroid eye disease is a rare and vision-threatening autoimmune condition that causes the muscles and fatty tissues behind the eye to become inflamed and enlarged, leading to bulging of the eyes. In addition to the bulging appearance, patients can experience double vision and light sensitivity. The disease can lead to blindness.

Teprotumumab is a fully human monoclonal antibody which blocks the inflammatory autoimmune pathophysiology that underlies thyroid eye disease. The U.S. Food and Drug Administration approved Horizon Therapeutics Tepezza (teprotumumab) in January as the first therapy for thyroid eye disease.

The randomized, double-blind, placebo-controlled trial was conducted by the department of surgery at Cedars-Sinai and at other medical centers nationwide.

“The study demonstrates that medical treatment with teprotumumab is effective at reversing the manifestations of disease, providing new hope for patients,” said the study’s principal investigator Raymond Douglas, director of the Orbital and Thyroid Eye Disease Program at Cedars-Sinai. Douglas is a consultant to Horizon Therapeutics, which manufactures the drug and funded the study.

Patients received the drug intravenously once every week for three weeks over a 21-week period. Patients who were administered teprotumumab experienced effective response in two doses or six weeks of administration.

After 24 weeks, the study showed 83 percent of people on the drug had measurable reduction in eye bulge compared to 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.

“Other than highly invasive surgical procedures, patients with thyroid eye disease had no real treatment alternatives,” Douglas said. “This is a medical breakthrough for a very large percentage of the patient population to receive an alternative medical infusion treatment with great results, quickly.”

Photo: Raymond Douglas, director of the Orbital and Thyroid Eye Disease Program at Cedars-Sinai

Author: Rare Daily Staff