Mirum’s CTEXLI™ (chenodiol) Tablets Receives FDA (U.S. Food and Drug Administration) Approval for Treatment of Cerebrotendinous Xanthomatosis (CTX). Ctexli is the first FDA-approved drug to treat CTX, a very rare lipid storage disease, in adults.
- CTEXLI is the first and only medication approved for the treatment of CTX in adults
- Approval based on Phase 3 RESTORE study results
- CTEXLI granted U.S. FDA Orphan Drug exclusivity for the treatment of CTX
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 (urine 23S-pentol) was highly statistically significant (p<0.0001). At the end of the randomized double-blind withdrawal period, there was a 20-fold difference between placebo and CTEXLI treated patients in urine 23S-pentol levels.
To read Mirum Pharmaceuticals press release: Mirum Pharma
To read the FDA press release: FDA Approves CTX treatment
To find out more about the treatment: CTEXLI™ (chenodiol)
