Perspective

Horizon files biologics license application for thyroid eye disease treatment

Horizon Therapeutics has submitted a biologics license application to the FDA for its investigational thyroid eye disease treatment candidate teprotumumab, according to a press release.

The application includes results from the OPTIC confirmatory phase 3 clinical trial, which compared teprotumumab infusion with placebo and measured improvement in proptosis as the primary endpoint. Proptosis improved 2 mm or more in 82.9% of treated patients compared with 9.5% in the placebo group.

“There is a major unmet need for an effective therapy for active thyroid eye disease, a painful and sight-threatening disease with no FDA-approved treatment options,” Timothy Walbert, chairman, president and CEO of Horizon said. “Our BLA submission is an important step toward our goal of making teprotumumab available as soon as possible for patients with this debilitating disease.”

Horizon has requested priority review of the application. Teprotumumab has breakthrough therapy, orphan drug and fast track designations from the FDA. A priority review could mean a 6-month review process.

 

Horizon Therapeutics has submitted a biologics license application to the FDA for its investigational thyroid eye disease treatment candidate teprotumumab, according to a press release.

The application includes results from the OPTIC confirmatory phase 3 clinical trial, which compared teprotumumab infusion with placebo and measured improvement in proptosis as the primary endpoint. Proptosis improved 2 mm or more in 82.9% of treated patients compared with 9.5% in the placebo group.

“There is a major unmet need for an effective therapy for active thyroid eye disease, a painful and sight-threatening disease with no FDA-approved treatment options,” Timothy Walbert, chairman, president and CEO of Horizon said. “Our BLA submission is an important step toward our goal of making teprotumumab available as soon as possible for patients with this debilitating disease.”

Horizon has requested priority review of the application. Teprotumumab has breakthrough therapy, orphan drug and fast track designations from the FDA. A priority review could mean a 6-month review process.

 

    Perspective
    Roger A. Dailey

    Roger A. Dailey

    It is very exciting that this potential first FDA-approved drug for the treatment of thyroid eye disease is moving along. It is certainly the biggest advancement in the treatment of thyroiditis in the last 36 years since I have been involved with management of these patients.

    We hope that teprotumumab could be available for us to use for our patients in the first quarter of 2020, given that it has the breakthrough, orphan drug and fast track designations from the FDA.

    The treatment is labeled for active disease, but we may be able to apply it earlier in the active phase rather than when disease is moderate to severe. That might enable us to keep patients from developing problems of proptosis, lid retraction and double vision that are so disabling and that require surgery down the road.

    • Roger A. Dailey, MD, FACS
    • Portland, Oregon

    Disclosures: Dailey reports he is a member of the Horizon advisory board.