Multi-Center Study Results Lead to FDA Approval of Novel Treatment for Thyroid Eye Disease

Feb 13, 2020 | Graves' Disease, Graves’ Orbitopathy, Thyroid Eye Disease

Multi-Center Study Results Lead to FDA Approval of Novel Treatment for Thyroid Eye Disease

Michigan Medicine, Cedars-Sinai, and other preeminent research centers recently concluded a study centering on what could possibly represent a future cure for thyroid eye disease. The Phase 2 clinical trial, assessing the drug teprotumumab, produced results that were recently published in New England Journal of Medicine. The study drug was recently approved by the U.S. Food and Drug Administration.

The Disease

Thyroid eye disease, also known as Graves’ Orbitopathy, is an autoimmune condition in which immune cells attack the thyroid gland which responds by secreting an excess amount of thyroid hormone. Consequently, the thyroid gland enlarges and excel hormones increase metabolism reports Michigan Medicine. The eyes are  particularly vulnerable to Graves’ eye disease because the autoimmune attack often seeks the eye muscles and connective tissue within the eye socket. Up to 20% of patients can have sight threatening disease. About one million Americans are diagnosed with the disease annually, reports Michigan Medicine.

The Drug:  Teprotumumab Just Approved by FDA

Sold under the brand name Tepezza, this medication is used to treat adults with thyroid eye disease  Acquired by Horizon Pharma from River Valley Development Corp, the drug binds to IGF-1R. It was approved in the United States by the FDA just in January 2020. See the label for more information including common side effects.

The study

The study sponsor and investigators sought to investigate the efficacy, safety, and tolerability of RV 001 (teprotumumab), a fully human anti-IGF1R antibody, administered q3W for 6 months, in comparison to placebo, in the treatment of participants suffering from active thyroid eye disease. The Phase II study, sponsored by Horizon Pharma, involved  dozens of research sites and was led by principal investigator Raymond Douglas from Cedar Sinai in Los Angeles. The results of the study were published in the New England Journal of Medicine. The study revealed that in patients with active ophthalmopathy, teprotumumab was more effective than placebo in reducing proptosis and the Clinical Activity Score


This study was funded by River Vision Development as well as a U.S. FDA Office of Orphan Products Development grants and other funding sources. Note, sponsor Horizon Pharma acquired the asset as part of its acquisition of River Vision Development Corp.

About Horizon Pharma

Ireland-based Horizon Pharma (Horizon) was founded in 2008 as a startup with only a handful of employees and no office space. They now have over 1,200 employees focusing on developing treatments for patients impacted by rare and rheumatic conditions. This Irish-tax registered company centers its model on the acquisition, rebranding and repackaging—as well as repricing—of specialist drugs for sale into America’s healthcare system which represents 97% of its worldwide sales by 2017. The company made its move to Ireland—in search of lower tax rates and overall friendlier corporate tax system in Ireland. The drug portfolio consists of treatments for arthritis, pain and inflammatory diseases. By 2016, the company became a member of the Pharmaceutical Research and Manufacturer s of America (PhRMA). By 2017 the company cross the $1 billion sales mark

Lead Research/Investigator

Terry J. Smith, MD, the Frederick G.L. Huetwell Professor in Ophthalmology and Visual Sciences at the University of Michigan

Raymond Douglas, MD, PhD

For other study authors, see the source link


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