Horizon Therapeutics has submitted a Biologics License Application (BLA) to the Food and Drug Administration (FDA) for teprotumumab 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). 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.
The BLA is 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. The primary end point of the OPTIC trial was the percentage of patients with a ≥2mm reduction of proptosis from baseline in the study eye without deterioration of proptosis in the non-study eye at study week 24.
Results showed a statistically significant proportion of patients receiving teprotumumab were proptosis responders at week 24 compared with placebo (82.9% [N=34/41] vs 9.5% [N=4/42]; P <.001). Moreover, all secondary end points were met in the study. Regarding safety, teprotumumab was generally well tolerated.
“There is a major unmet need for an effective therapy for active thyroid eye disease, a painful and sight-threatening disease with no FDA-approved treatment options,” said Timothy Walbert, chairman, president and CEO, Horizon. “Our BLA submission is an important step toward our goal of making teprotumumab available as soon as possible for patients with this debilitating disease.”
Teprotumumab was previously granted Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA. The Company has requested priority review for the BLA.
For more information visit horizontherapeutics.com.