Horizon Therapeutics plc submits teprotumumab BLA to FDA for the treatment of active thyroid eye disease

Horizon Therapeutics plc announced that it has submitted a Biologics License Application (BLA) to the FDA for its investigational medicine teprotumumab for the treatment of active thyroid eye disease (TED). Teprotumumab has Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA. Horizon requested priority review for the application, which, if granted, could result in a six-month review process. The FDA has a 60-day filing review period to determine whether the BLA is complete and acceptable for filing. The teprotumumab BLA submission includes results from the Phase III confirmatory clinical trial, called OPTIC (Treatment of Graves’ Orbitopathy (Thyroid Eye Disease) to Reduce Proptosis with Teprotumumab Infusions in a Randomized, Placebo-Controlled, Clinical Study), as well as positive Phase II results. Results from OPTIC were presented at the 2019 American Association of Clinical Endocrinologists (AACE) Scientific and Clinical Congress.

The study met its primary endpoint, showing that significantly more patients treated with teprotumumab compared with placebo had a meaningful improvement in proptosis, or bulging of the eye: 82.9 percent of teprotumumab patients compared to 9.5 percent of placebo patients achieved the primary endpoint of a 2 mm or more reduction in proptosis (p<0.001). All secondary endpoints were also met.