Horizon Therapeutics plc announced positive and statistically significant topline results from its randomized, double-masked, placebo-controlled Phase 4 clinical trial evaluating TEPEZZA for the treatment of adults with chronic TED and low CAS, which is a measure of disease activity. The totality of clinical trial data continues to strongly support the efficacy of TEPEZZA across a broad spectrum of TED patients regardless of disease activity or duration, with a well-established safety profile. TEPEZZA is the first and only medicine approved by the U.S. Food and Drug Administration (FDA) for the treatment of TED – a serious, progressive, debilitating and potentially vision-threatening rare autoimmune disease.
The Phase 4 trial evaluated patients with an initial diagnosis of TED between two to 10 years and low levels of disease activity, whereas the prior pivotal trials that formed the basis of the original FDA approval of TEPEZZA evaluated patients with disease duration of nine months or less and higher levels of disease activity.
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“We are thrilled with the topline results, which reinforce that TEPEZZA significantly reduces proptosis in people living with Thyroid Eye Disease, regardless of their disease activity or duration, and underscores what we learned from our initial trials and what we have seen through more than three years of real-world use of TEPEZZA,” said Elizabeth H.Z. Thompson, Ph.D., executive vice president, research and development, Horizon. “With TEPEZZA, physicians have a medicine that can be used in a broad range of Thyroid Eye Disease patients, including those with long-duration disease of more than 5 years on average in this trial, which is important because we know the negative impact of the disease can be significant across all types of Thyroid Eye Disease patients. We look forward to discussing these data with the FDA to determine our next steps.”
Topline Data Overview
At Week 24, topline data per the pre-specified primary analysis method (intent-to-treat) demonstrated that the primary endpoint was met, and patients treated with TEPEZZA achieved a statistically significant reduction in proptosis from baseline compared to those receiving placebo. In addition, in the pre-specified per-protocol analysis, the differences between patients treated with TEPEZZA and patients treated with placebo increased.
SOURCE: Businesswire