FDA Advisory Committee Votes Unanimously to Support the Use of Teprotumumab for the Treatment of Thyroid Eye Disease (TED)
FDA Advisory Committee Votes Unanimously to Support the Use of Teprotumumab for the Treatment of Thyroid Eye Disease (TED)
-- If approved, teprotumumab would be the first and only approved medicine for people living with TED, fulfilling a significant unmet need --
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
“Today’s unanimously positive vote marks a significant step towards the first
“TED can affect patients both physically and emotionally, limiting their ability to perform everyday activities like driving, working, reading, sleeping and participating in social activities,” said
The
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
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
About Teprotumumab
Teprotumumab is a fully human monoclonal antibody (mAb) and a targeted inhibitor of the insulin-like growth factor 1 receptor (IGF-1R). Teprotumumab has received Priority Review, Orphan Drug, Fast Track and Breakthrough Therapy designations from the
About Horizon
Horizon is focused on researching, developing and commercializing medicines that address critical needs for people impacted by rare and rheumatic diseases. Our pipeline is purposeful: we apply scientific expertise and courage to bring clinically meaningful therapies to patients. We believe science and compassion must work together to transform lives. For more information on how we go to incredible lengths to impact lives, please visit www.horizontherapeutics.com, follow us @HorizonNews on Twitter, like us on
Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding the timing of an
References
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Journal of Ophthalmology . 2015. https://www.hindawi.com/journals/joph/2015/249125/cta/. AccessedFeb 22, 2019 . -
Ross DS, et al. The 2016
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Shan SJ, Douglas RS. The Pathophysiology of Thyroid Eye Disease.
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The New England Journal of Medicine .25 February 2010 . https://www.nejm.org/doi/full/10.1056/NEJMra0905750. AccessedFeb 22, 2019 . -
Pritchard J, et al. Igs from patients with Graves' disease induce the expression of T cell chemoattractants in their fibroblasts.
The Journal of Immunology .15 January 2002 . https://www.ncbi.nlm.nih.gov/pubmed/11777993. AccessedFeb 22, 2019 . -
McKeag D, et al. Clinical features of dysthyroid optic neuropathy: a
European Group on Graves' Orbitopathy (EUGOGO) survey.British Journal of Ophthalmology .11 October 2006 . https://www.ncbi.nlm.nih.gov/pubmed/17035276. AccessedFeb 22, 2019 . -
Mamoojee Y, Pearce SHS. Natural History. In: Wiersinga WM, Kahaly GJ (eds): Graves’ Orbitopathy: A Multidisciplinary Approach – Questions and Answers.
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Bothun ED, et al. Update on thyroid eye disease and management. Clinical Ophthalmology.
19 October 2009 . https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2770865/. AccessedFeb 22, 2019 .
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