FDA Updates Approval Language for Horizon TED Drug Tepezza

Rare Daily Staff

The U.S. Food and Drug Administration approved an update to the Indications and Usage section of the Tepezza label to specify its use for the treatment of thyroid eye disease regardless of activity or duration.

Tepezza is the first and only medicine approved by the FDA for the treatment of thyroid eye disease (TED), a serious, progressive, debilitating and potentially vision-threatening rare autoimmune disease.

TED often occurs in people living with Graves’ disease, but it is a distinct disease that is caused by autoantibodies activating an IGF-1R-mediated signaling complex on cells within the retro-orbital space. This leads to a cascade of negative effects, which may cause long-term, irreversible damage, including blindness. Signs and symptoms of TED may include dry eyes and grittiness; redness, swelling and excessive tearing; eyelid retraction; proptosis; pressure and/or pain behind the eyes and diplopia.

Tepezza is the first and only medicine approved by the FDA for the treatment of TED. It is a fully human monoclonal antibody and a targeted inhibitor of the insulin-like growth factor-1 receptor (IGF-1R) that is administered to patients once every three weeks for a total of eight infusions.

The label update follows positive topline results from a randomized, double-masked, placebo-controlled phase 4 clinical trial that demonstrated that patients with an initial diagnosis of TED between 2 to 10 years (mean duration of 5.2 years) and with low disease activity achieved a statistically significant reduction in proptosis from baseline at Week 24 after receiving Tepezza compared to those receiving placebo. In the trial, no new safety signals were observed.

“While Tepezza already had a broad indication for the treatment of thyroid eye disease, the new indication language now references treatment regardless of disease activity or duration,” said Tim Walbert, chairman, president, and CEO of Horizon. “The updated indication reinforces the importance of unrestricted access for all eligible patients across the full spectrum of thyroid eye disease. This creates an opportunity to ease the access burden for patients and physicians with the goal of decreasing time to therapy for patients who may benefit from Tepezza.”

Photo: Tim Walbert, chairman, president, and CEO of Horizon