After years without treatment options, people living with thyroid eye disease got a surprise from the FDA this week.
Well before its anticipated March decision, the agency approved a first-of-its-kind medicine to treat the autoimmune disease. The drug, teprotumumab, is administered by infusion.
Wiley Chambers, MD, of the FDA’s Center for Drug Evaluation and Research called the approval an “important milestone” for thyroid eye disease. He also noted that it, “represents important progress in the approval of effective treatments for rare diseases.”
Thyroid eye disease often occurs in people with Graves’ disease, who suffer from overactive thyroid. Muscles behind the eye become inflamed, causing the eye to bulge. Misalignment and double vision can result, and the disease may lead to blindness. Like other vision conditions, thyroid eye disease has a multifaceted impact. As the disease progresses, people may find themselves unable to perform work duties, engage in social situations and manage the basics of independent living.
The FDA’s early approval reflects both the disease’s impact and the unmet need among people with thyroid eye disease. Previous treatment approaches were limited, even invasive. Patients relied on radiation, corticosteroids to reduce swelling, and bone-shaving surgeries.
At the prospect of a treatment specifically for thyroid eye disease, the FDA granted teprotumumab priority review and fast tracked its approval process. And when the agency’s Dermatologic and Ophthalmic Drugs Advisory Committee considered the drug last month, committee members voted unanimously in favor of approval. The newly approved drug is designated a breakthrough therapy.
Innovators and regulators have worked in tandem to provide thyroid eye disease patients with their first real chance at effective treatment. It now falls to health plans to make the drug accessible to those who need it.