The Food and Drug Administration (FDA) has accepted for priority review the Biologics License Application (BLA) for teprotumumab (Horizon Therapeutics) for the treatment of active thyroid eye disease. 

Teprotumumab is an investigational fully human monoclonal antibody that works by inhibiting the insulin-like growth factor 1 receptor (IGF-1R). The BLA was supported by data from the randomized, placebo controlled, phase 3 confirmatory OPTIC trial, which evaluated the safety and efficacy of teprotumumab in 83 patients with moderate to severe thyroid eye disease, as well as positive results from a phase 2 trial.

A Prescription Drug User Fee Act date of March 8, 2020 has been set for the application. “The accelerated review timeline is particularly important given that there is no FDA-approved medical treatment for [thyroid eye disease] and the window of time for treatment is limited before patients experience potentially long-term, permanent damage to their eyes,” said Timothy Walbert, chairman, president and chief executive officer, Horizon.

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Thyroid eye disease is an autoimmune condition that is caused by autoantibodies activating an IGF-1R-mediated signalling complex, thus resulting in inflammation and tissue expansion behind the eye. Disease progression can lead to proptosis, strabismus, diplopia, and potentially blindness. 

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