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FDA recommends approval of eye drug tested in Portland

OHSU is part of multi-center study testing teprotumumab for thyroid eye disease
Thyroid eye disease treatment
Judy Bachman's eyes are measured for bulging during an appointment. Bachman participated in a clinical trial that treated symptoms of thyroid eye disease, a condition that can cause eyes to bulge outward, double vision, and eyelids to retract and not fully close. (OHSU/Kristyna Wentz-Graff)

UPDATE: On Jan. 21, 2020, the FDA approved teprotumumab’s use to treat thyroid eye disease. More information is available in the FDA news release.

A federal committee’s recommendation that a new drug be approved to treat a rare, but debilitating eye condition could mean more people like Judy Bachman get their lives back.

“My eyes were protruding more and more each day and had become misaligned. I was seeing double and could not track objects I was trying to focus on,” said Bachman, of Portland, who received infusions of the drug teprotumumab at Oregon Health & Science University as part of the drug’s phase 2 clinical trial in 2015 and 2016. She testified before the committee about her experience with the drug through the trial.

“I stopped reading because I couldn’t track the words. Sewing was torture, and I couldn’t go bicycling,” Bachman continued. “But I don’t have those troubles now. Those tasks are so simple now, compared to what they were before the clinical trial.”

The Food and Drug Administration’s Dermatologic and Ophthalmic Drugs Advisory Committee recommended after its Dec. 13 hearing to allow teprotumumab to be prescribed for patients with thyroid eye disease, which can cause eyes to bulge outward, eyelids to retract and not fully close, double vision and more. The FDA is expected to make a final decision by March 2020.

Thyroid eye disease can lead to blindness if untreated. Also known as Graves’ eye disease, the autoimmune condition is often associated with thyroid disorders and is believed to affect up to 75,000 Americans.

Thyroid eye disease treatment
Bachman and Dr. Dailey recently testified before the FDA the benefits of the medication. (OHSU/Kristyna Wentz-Graff)

Until now, there hasn’t been an approved drug to treat the condition itself. Existing treatments only address symptoms, but not the condition’s root cause: overactivation of insulin-like growth factor 1 receptors, which causes inflammation and tissue expansion behind the eye.

“Patient self-confidence is destroyed by this disease,” said Roger Dailey, M.D., FACS, a professor of ophthalmology in OHSU’s School of Medicine and Casey Institute who led OHSU’s involvement in the clinical trial and also testified before the committee. “Unkind people ridicule patients with the condition, and they often have difficulty finding or maintaining employment. Having a drug that reduces the disfiguring signs and visually disturbing symptoms can absolutely dramatically improve a patient’s quality of life.”

Of the participants who completed the drug’s Phase 2 clinical trial, nearly 69% experienced reduced bulging of their eye by at least 2 mm. In Phase 3, the drug company sponsoring the trial reported that same result increased to nearly 83% of trial participants. Before the trial, only surgery could reduce eye bulging, also known as proptosis.

Horizon Therapeutics led the drug’s clinical trials and requested FDA approval of its use for thyroid eye disease.

This research was supported in part by the National Center for Advancing Translational Sciences of the National Institutes of Health under award number UL1TR0002369, as well as an unrestricted grant from Research to Prevent Blindness.

In the interest of ensuring the integrity of our clinical care practices and as part of our commitment to public transparency, OHSU actively regulates, tracks and manages relationships that our clinicians may hold with entities outside of OHSU. In regards to this drug, Dr. Dailey has received payments for consulting from Horizon Therapeutics. Review details of OHSU's conflict of interest program to find out more about how we manage these business relationships.

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