Horizon Therapeutics plc announced that the U.S. Food and Drug Administration (FDA) has 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 thyroid eye disease activity or duration.” Tepezza is the first and only medicine approved by the FDA for the treatment of TED—a serious, progressive, debilitating and potentially vision-threatening rare autoimmune disease.
The label update follows positive topline results from a randomized, double-masked, placebo-controlled phase 4 clinical trial (NCT04583735) that were announced lastweek, which demonstrated that patients with an initial diagnosis of TED between 2 to 10 years (mean duration of 5.2 years; SD 1.77) and with low disease activity, or CAS, (mean CAS of 0.4; SD 0.49) 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.
“We worked closely with the FDA on this important label update, which further reinforces the potential benefit of Tepezza in people impacted by [TED] regardless of disease activity or duration,” said Tim Walbert, chairman, president and chief executive officer, Horizon. “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. 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.”
The Company plans to present data from the phase 4 trial at a future medical congress and publish the data in a peer-reviewed medical journal.