Horizon Therapeutics plc yesterday announced that the U.S. Food and Drug Administration (FDA) has approved TEPEZZATM (teprotumumab-trbw) for the treatment of Thyroid Eye Disease (TED). Horizon reports that TEPEZZA is the first and only FDA-approved medicine for the treatment of TED, a rare, vision-threatening autoimmune disease associated with proptosis, diplopia, blurred vision, pain, inflammation and facial disfigurement. The drug is a fully human monoclonal antibody (mAb) and a specific inhibitor of the insulin-like growth factor-1 receptor (IGF-1R) that is administered to patients once every three weeks for a total of eight infusions.
The FDA approval of TEPEZZA comes ahead of the Prescription Drug User Fee Act (PDUFA) goal date of March 8, 2020. The medicine received Priority Review, Orphan Drug, Fast Track and Breakthrough Therapy designations from the FDA.
The FDA approval of TEPEZZA is supported by positive results from the Phase 2 clinical study, as well as the Phase 3 confirmatory clinical study OPTIC (Treatment of Graves’ Orbitopathy [Thyroid Eye Disease] to Reduce Proptosis with Teprotumumab Infusions in a Randomized, Placebo-Controlled, Clinical Study). The OPTIC study found that significantly more patients treated with TEPEZZA (82.9%) had a meaningful improvement in proptosis as compared with placebo without deterioration in the fellow eye at Week 24. Additional secondary endpoints were also met, including a change from baseline of at least one grade in diplopia in 67.9% of patients receiving TEPEZZA compared to 28.6% of patients with placebo. Analysis of the Phase 2 and Phase 3 clinical studies further found more patients with complete resolution of diplopia among those treated with TEPEZZA (53%) compared with those treated with placebo (25%).
The majority of adverse events experienced with TEPEZZA treatment were graded as mild to moderate and were manageable in the trials, with few discontinuations or therapy interruptions. In addition, Horizon will conduct a post-marketing study to evaluate both safety in a larger patient population and retreatment rates relative to how long patients receive the medicine.
TEPEZZA is expected to be available in the United States in the coming weeks.
Full press release: https://ir.horizontherapeutics.com/news-releases/news-release-details/fda-approves-tepezzatm-teprotumumab-trbw-treatment-thyroid-eye
Source: Horizon Therapeutics