India, Feb. 24 -- Mirum Pharmaceuticals, Inc. (MIRM) Monday announced that the U.S. Food and Drug Administration (FDA) has approved CTEXLI (chenodiol) tablets, a bile acid, for the treatment of adults with cerebrotendinous xanthomatosis (CTX).
CTEXLI is 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 evaluating the safety and efficacy of CTEXLI in adult patients with CTX by measurement of urine bile alcohols and other secondary measures. The primary endpoint of reduction in bile alcohols was highly statistically significant. At the end of the randomized double-blind withdrawal period, there was a 20-fold difference between placebo and C...