According to a story from Biospace, the drug company Horizon Therapeutics plc has recently announced that the US Food and Drug Administration (FDA) has approved the company’s drug teprotumumab-trbw (marketed as TEPEZZA™) as a treatment for thyroid eye disease (TED). This is a historic moment for this rare disease community as the news marks the first ever FDA approved treatment for thyroid eye disease.
About Thyroid Eye Disease
Thyroid eye disease, which is also called Graves’ ophthalmopathy, is a rare, inflammatory disorder that affects the orbital and periorbital tissues around the eye. People with this condition often have eyes that are abnormally bulged in appearance. It is an autoimmune disease, meaning that it is triggered by an abnormal immune response in which the system mistakenly attacks healthy tissue. While most common in people with Graves’ disease, the condition can appear alongside a variety of conditions that affect the thyroid. Women are more likely to be affected than men. Symptoms include conjunctivitis, retracted upper eyelids, swelling, eye redness, lid lag, and bulging eyes. Though some cases are mild and resolve on their own, more serious cases constitute a medical emergency due to compression of the optic nerve and should be treated promptly. Treatment includes steroids and other medication to bring down inflammation, surgery, and medications to regulate thyroid hormones. To learn more about thyroid eye disease, click here.
TEPEZZA is categorized as a monoclonal antibody and is an inhibitor of IGF-1R, or the insulin-like growth factor-1 receptor. The drug produced positive results in clinical testing. The drug was able to have a significant impact on proptosis (eye bulging) in 82.9 percent of patients that tried it. TEPEZZA also cause a diverse array of secondary improvements as well, such as improvements in double vision, which occurred in 67.9 percent of users. These two symptoms are among the most challenging aspects of thyroid eye disease for many patients.
TEPEZZA gained recognition from the FDA for its effectiveness during development, earning Orphan Drug, Fast Track, and Breakthrough Therapy designations, as well as Priority Review. Horizon will continue to study the drug in a large scale trial that will evaluate the long term safety of the therapy in a larger patient population.
Ultimately, this approval is of great significance to the thyroid eye disease patient community, who will now have the opportunity to see improvements with a therapy that is precisely tailored to their illness.