FDA Committee Unanimously Recommends Approval of Teprotumumab for Thyroid Eye Disease

The Dermatologic and Ophthalmic Drugs Advisory Committee of the FDA voted 12-0 Friday in favor of recommending approval of a biologics license application for teprotumumab, an experimental human monoclonal antibody shown to dramatically reduce the most debilitating symptoms of Graves’ orbitopathy.

Committee members expressed concern before casting votes about the small patient population studied — fewer than 90 adults treated with infusions of teprotumumab (Horizon Therapeutics) have been enrolled in controlled clinical trials — and several adverse events reported, among them hearing loss, muscle spasms, hyperglycemia and infections. In briefing materials, the FDA noted the population was considerably smaller than the common safety database of greater than 300 patients treated with a course of therapy. However, committee members ultimately agreed that the benefits of the drug outweighed any potential risks.