FDA Advisory Committee Votes Unanimously to Support the Use of Teprotumumab for the Treatment of Thyroid Eye Disease (TED) - Seite 2

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 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.

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.

The OPTIC study was initiated after the Phase 2 study demonstrated clinically meaningful and highly statistically significant results in reducing proptosis and in the symptoms of TED as measured by Overall Treatment Response. The Phase 2 study was published in The New England Journal of Medicine in May 2017. The Phase 2 and Phase 3 studies form a robust body of clinical evidence on the use of teprotumumab in people living with TED.

About Thyroid Eye Disease

Thyroid Eye Disease (TED) is a serious, progressive and vision-threatening autoimmune disease with a limited window of activity that can last up to three years.^1,2,3 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 orbit.^4,5 This leads to a cascade of negative effects, which may cause long-term, irreversible damage. Active TED lasts for up to three years and is characterized by inflammation and tissue expansion behind the eye.^1,6 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.^2,7 TED has only been shown to respond to pharmacotherapy while the disease is active and inflammation is ongoing.⁸ Currently, patients must live with active TED until the inflammation subsides, after which they are often left with permanent and vision-impairing consequences.^1,6