Horizon Therapeutics announced the U.S. FDA has approved Tepezza (teprotumumab-trbw) for the treatment of Thyroid Eye Disease (TED). Tepezza is the first and only FDA-approved medicine for the treatment of TED. The FDA approval of Tepezza comes ahead of the Prescription Drug User Fee Act (PDUFA) goal date of March 8, 2020.
The FDA approval of Tepezza was based on results from a Phase 2 clinical study, as well as the Phase 3 confirmatory clinical study, OPTIC.
The phase 2 study, the results of which were published in The New England Journal of Medicine in May of 2017, was multicenter, double-masked, randomized, placebo-controlled trial to determine the efficacy and safety of teprotumumab in patients with active, moderate-to-severe ophthalmopathy. A total of 88 patients were randomly assigned to receive placebo or active drug administered intravenously once every 3 weeks for a total of eight infusions. The primary end point was the response in the study eye. This response was defined as a reduction of 2 points or more in the Clinical Activity Score (scores range from 0 to 7, with a score of ≥3 indicating active thyroid-associated ophthalmopathy) and a reduction of 2 mm or more in proptosis at week 24. In the intention-to-treat population, 29 of 42 patients who received teprotumumab (69%), as compared with 9 of 45 patients who received placebo (20%), had a response at week 24.
The phase 3 OPTIC study was a randomized, double-masked, placebo-controlled, parallel-group, multicenter study enrolled 83 subjects with moderate-to-severe active TED. Significantly more patients treated with Tepezza (82.9%) had a meaningful improvement in proptosis (≥ 2 mm) as compared with placebo patients (9.5%) without deterioration in the fellow eye at Week 24.
In a related analysis of the Phase 2 and Phase 3 clinical studies, there were 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 related to Tepezza included muscle spasm, nausea, alopecia, diarrhea, fatigue, hyperglycemia, hearing impairment, dry skin, dysgeusia and headache.
Tepezza was previously granted Priority Review, Orphan Drug, Fast Track and Breakthrough Therapy designations from the FDA.
About Tepezza (teprotumumab-trbw)
Tepezza is an insulin-like growth factor-1 receptor inhibitor. Teprotumumab-trbw binds to IGF-1R and blocks its activation and signaling.
About Thyroid Eye Disease
Thyroid Eye Disease (TED) is a serious, progressive and vision-threatening rare autoimmune disease. While TED often occurs in people living with hyperthyroidism or Graves’ disease, it is a distinct disease that is caused by autoantibodies activating an IGF-1R-mediated signaling complex on cells within the retro-orbital space. This leads to a cascade of negative effects, which may cause long-term, irreversible damage. As TED progresses, it causes serious damage – including proptosis (eye bulging), strabismus (misalignment of the eyes) and diplopia (double vision) – and in some cases can lead to blindness. Historically, patients have had to live with TED until the inflammation subsides, after which they are often left with permanent and vision-impairing consequences.