Adults with thyroid eye disease may be able to prevent blindness with the use of teprotumumab. Teprotumumab which was approved by the U.S. Food and Drug Administration on Tuesday is the first approved drug specifically meant for the treatment of thyroid eye disease. The marketing of the drug will be done under the brand name Tepezza.
The lead author of one of the studies that informed the FDA’s decision, Dr. Raymond Douglas, said that the new FDA approved drug has the potential to offer a new approach and a paradigm shift. “Right now, we use supportive therapy for patients with thyroid eye disease, but there is often no significant improvement. Instead, the symptoms decrease the quality of their life. Teprotumumab, on the other hand, was extraordinarily effective. We hope that the drug eliminates the suffering of people with the disease and allow them to live a better life. With treatment, they will not need to worry about going blind or becoming disfigured,” Dr. Douglas stated.
Thyroid eye disease is a rare condition that affects adults and has the potential to cause blindness. Patients with this condition often have bulgy eyes caused by inflammation in the back of the eye. The swelling may result in the squeezing of the optic nerve and when this happens, the patient may not be able to see clearly.
Everyone with an overactive thyroid gland caused by Graves’ disease is susceptible to this eye disease. Often, the disease results in redness of the eye, swelling, double vision, dryness of the eye or excessive tearing, and sensitivity to light. The patients may also feel like they have particles in their eyes all the time. The most noticeable symptom of the disease is bulging of the eyes.
Sometimes, it may even be difficult for people with the disease to completely shut their eyes. The new FDA approved drug was given an “orphan drug” status because the disease is rare. The status was to encourage interested pharmaceutical companies to develop drugs for the treatment of the disease. Apart from giving incentives to these companies, the FDA gave the drug a priority review as well as fast track and breakthrough therapy designations. All of these were done to help the manufacturer get the new drug into the market.
The FDA made its decision to approve teprotumumab after the findings of two studies that included 170 active thyroid eye disease patients. The last study which was led by Douglas was a phase 3 clinical trial that involved more than 80 people with the disease. These patients were randomly assigned to receive either a placebo or teprotumumab. Patients received treatment once every week for three weeks and teprotumumab was administered intr
Dr. Douglas reports that the FDA approved drug started working after just two doses. By week 24, 83% of those who participated in the clinical trial and received the drug experienced a 2-millimeter reduction in proptosis (eye protrusion). Only 10% of people on placebo reported a reduction in proptosis. Douglas said that teprotumumab reversed double vision and had minimal side effects. The most common side effects are nausea, hair loss, fatigue, and muscle cramps. Pregnant women are, however, advised not to use the drug.