Horizon Therapeutics 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 6-month review process. The FDA has a 60-day filing review period to determine whether the BLA is complete and acceptable for filing.
“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,” Timothy Walbert, chairman, president and chief executive officer, Horizon, said in a company news release. “Our BLA submission is an important step toward our goal of making teprotumumab available as soon as possible for patients with this debilitating disease.”
The teprotumumab BLA submission includes results from the phase 3 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 2 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.
Teprotumumab was generally well tolerated. The majority of adverse events were mild or moderate, manageable and resolved during or after treatment. The confirmatory OPTIC study was initiated after clinically meaningful and highly statistically significant results from a Phase 2 study, published in The New England Journal of Medicine on May 4, 2017. Teprotumumab is an investigational medicine and its safety and efficacy have not been established.