FDA Advisory Committee voted unanimously for teprotumumab to treat thyroid eye disease . -Horizon Therapeutics

Horizon Therapeutics plc announced that the Dermatologic and Ophthalmic Drugs Advisory Committee (DODAC) of the FDA voted unanimously that the potential benefits of teprotumumab, a fully human monoclonal antibody (mAb), outweigh the potential risks for the treatment of Thyroid Eye Disease (TED). TED is a rare, serious, progressive and vision-threatening autoimmune disease, and is associated with proptosis (eye bulging), diplopia (double vision), blurred vision, pain and facial disfigurement that can significantly impact patients� quality of life. If approved, teprotumumab would be the first FDA-approved medicine for TED.

Teprotumumab is an investigational medicine and its safety and efficacy have not been established. The FDA is currently evaluating a Biologics License Application (BLA) for teprotumumab for the treatment of TED. Teprotumumab is one of less than five medicines ever to receive Priority Review, Orphan Drug, Fast Track and Breakthrough Therapy designations from the FDA. The Prescription Drug User Fee Act (PDUFA) action date is March 8, 2020. The FDA will consider today�s vote as it reviews the BLA, although it is not obligated to follow the Committee�s recommendation.

The efficacy and safety of teprotumumab for the treatment of TED is supported by a robust body of clinical evidence. The BLA submission is based on positive 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. The OPTIC study found that significantly more patients treated with teprotumumab had a meaningful improvement in proptosis, or bulging of the eye, as compared with placebo (82.9% of teprotumumab patients compared to 9.5% of placebo patients). These results were achieved within a six-month course of therapy. All secondary endpoints were also met, including reduced diplopia (double vision), improved quality of life (QoL) and reductions in Clinical Activity Score (CAS), which measures the degree of inflammation, including pain, swelling and redness. As previously reported, the majority of adverse events experienced with teprotumumab treatment were graded as mild to moderate and were managed in the trials, with few discontinuations.