Horizon Phase III teprotumumab data tee up BLA

Horizon Pharma plc (NASDAQ:HZNP) said teprotumumab (HZN-001) met the primary and all secondary endpoints in the Phase III OPTIC trial to treat Graves orbitopathy, also known as thyroid eye disease. The company plans to submit a BLA to FDA mid-year for the human mAb targeting insulin-like growth factor-1 receptor (IGF1R; CD221).

Thyroid eye disease is a progressive autoimmune disease in which IGF1R is overexpressed on orbital fibroblasts and can lead to proptosis (eye bulging), misalignment of the eyes, double vision and blindness. There are no FDA-approved therapies for the indication, which affects 15,000-20,000 patients each year in the U.S., according to Horizon...