Mirum Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved CTEXLI (chenodiol) tablets for the treatment of adults with cerebrotendinous xanthomatosis (CTX). This marks CTEXLI as the first and only treatment approved for this rare, progressive, and debilitating disease.
The approval is based on data from the Phase 3 RESTORE study, which evaluated the safety and efficacy of CTEXLI. The study demonstrated a highly statistically significant reduction in urine bile alcohols (p<0.0001) and improvements in serum cholestanol levels, which are key drivers of disease progression.
This regulatory milestone provides a crucial treatment option for patients with CTX, a condition where the body cannot properly break down cholesterol, leading to toxic accumulation and irreversible neurological dysfunction. The approval allows Mirum to actively promote CTEXLI in a market with high unmet medical need.
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