Horizon Therapeutics plc Announces the FDA has Granted Priority Review of the Teprotumumab Biologics License Application (BLA) for the Treatment of Active Thyroid Eye Disease (TED)
Horizon Therapeutics plc Announces the FDA has Granted Priority Review of the Teprotumumab Biologics License Application (BLA) for the Treatment of Active Thyroid Eye Disease (TED)
-- If Approved, Teprotumumab Would be the First FDA-Approved Medicine for this Vision-Threatening Disease --
“Priority Review for the teprotumumab BLA is another positive step toward our goal to make a difference in the lives of people who are living with active TED – a painful, debilitating and vision-threatening rare disease,” said
The
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The accepted BLA for teprotumumab includes 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). All secondary endpoints were also met, including reduced diplopia (double vision), improved quality of life (QoL) and reductions in Clinical Activity Score (CAS). Teprotumumab was generally well tolerated; the majority of adverse events were mild or moderate, manageable and resolved during or after treatment. Results of the OPTIC study were presented during the 2019
About 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.2,3,4 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.5,6 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.7,2 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.3,8 TED has only been shown to respond to pharmacotherapy while the disease is active and inflammation is ongoing.9 Currently, patients must live with active TED until the disease becomes inactive – often left with permanent and vision-impairing consequences.7,2
About Teprotumumab
Teprotumumab is a fully human monoclonal antibody (mAb) and a targeted inhibitor of the insulin-like growth factor 1 receptor (IGF-1R). By targeting the IGF-1R signal, teprotumumab reduces inflammation and prevents excessive muscle and fat cell expansion behind the eye. 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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Food and Drug Administration Amendments Act of 2007. https://www.congress.gov/bill/110th-congress/house-bill/3580. Accessed
Aug. 29, 2019 . -
Graves’ Ophthalmopathy: VISA versus EUGOGO Classification, Assessment, and Management.
Journal of Ophthalmology . 2015. https://www.hindawi.com/journals/joph/2015/249125/cta/. AccessedSept. 4, 2019 . -
The 2016
European Thyroid Association /European Group on Graves' Orbitopathy Guidelines for the Management of Graves' Orbitopathy.European Thyroid Journal .March 2016 . https://www.ncbi.nlm.nih.gov/pubmed/27099835. AccessedSept. 4, 2019 . -
Shan SJ, Douglas RS. The Pathophysiology of Thyroid Eye Disease.
Journal of Neuro-Ophthalmology . 2014; 34: 177-185. -
Graves' Ophthalmopathy.
The New England Journal of Medicine .Feb. 25, 2010 . https://www.nejm.org/doi/full/10.1056/NEJMra0905750. AccessedSept. 4, 2019 . -
Igs from patients with Graves' disease induce the expression of T cell chemoattractants in their fibroblasts.
The Journal of Immunology .Jan. 15, 2002 . https://www.ncbi.nlm.nih.gov/pubmed/11777993. AccessedSept. 4, 2019 . -
Update on thyroid eye disease and management. Clinical Ophthalmology.
Oct. 19, 2009 . https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2770865/. AccessedSept. 4, 2019 . -
Clinical features of dysthyroid optic neuropathy: A European Group on Graves' Orbitopathy (EUGOGO) survey.
British Journal of Ophthalmology .Oct. 11, 2006 . https://www.ncbi.nlm.nih.gov/pubmed/17035276. AccessedSept. 4, 2019 . -
Mamoojee Y, Pearce SHS. Natural History. In: Wiersinga WM, Kahaly GJ (eds): Graves’ Orbitopathy: A Multidisciplinary Approach – Questions and Answers.
Basel , Karger. 2017:93-104.
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Source:
Tina Ventura
Senior Vice President, Investor Relations
Investor-relations@horizontherapeutics.com
Ruth Venning
Executive Director, Investor Relations
Investor-relations@horizontherapeutics.com
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