FDA Greenlights First Treatment for Rare Autoimmune Eye Disease

An antibody drug developed by the Irish biopharma Horizon Therapeutics has been approved by the FDA for the treatment of adults with the rare autoimmune condition thyroid eye disease.

The approval of the drug came ahead of schedule, with the decision originally expected in March. The drug, a monoclonal antibody, is branded as Tepezza.

Thyroid eye disease, also known as Graves’ ophthalmopathy, is an autoimmune condition where antibodies inflame muscles behind the eye, causing bulging of the eyes. This can lead to eye pain and double vision. The condition is often seen in people with autoimmune diseases targeting the thyroid gland, such as Graves’ disease. Prior to Horizon Therapeutics’ Tepezza, there was no drug approved for thyroid eye disease.

“This treatment has the potential to alter the course of the disease, potentially sparing patients from needing multiple invasive surgeries by providing an alternative, non‑surgical treatment option,” stated Wiley Chambers, Deputy Director of the FDA’s Division of Transplant and Ophthalmology Products.

Tepezza is designed to treat thyroid eye disease by inhibiting a protein called insulin-like growth factor-1. This protein contributes to the inflammation in the eye muscles in thyroid eye disease, so blocking it can reduce the bulging of the eyes. The FDA’s approval was based on results from a phase III trial announced in 2019, where 83% of patients given the drug showed reduced bulging of the eyes, compared to just 10% of placebo patients.

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