NDAORALTABLETPriority Review
Approved
Feb 2025
Lifecycle
Growth
Competitive Pressure
30/100
Data completeness
4/8 — Partial
Priority Review
CTEXLI (chenodiol) by Mirum Pharmaceuticals is (chenodeoxycholic acid) is a primary bile acid, synthesized from cholesterol in the liver. Approved for cerebrotendinous xanthomatosis (ctx) in adults. First approved in 2025.
Drug data last refreshed 4h ago · AI intelligence enriched 6d ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
CTEXLI (chenodiol) is an oral bile acid replacement therapy approved for cerebrotendinous xanthomatosis (CTX), a rare genetic disorder caused by deficiency in sterol 27-hydroxylase. It restores endogenous chenodeoxycholic acid levels, activating farnesoid X receptor signaling to suppress atypical bile acids and reduce disease-associated neurological and systemic complications.
Growth5.7 years to LOE
Recently approved with growth trajectory ahead; commercial team will expand to build awareness in rare disease community and establish market presence against established competitor.
Mechanism of Action
(chenodeoxycholic acid) is a primary bile acid, synthesized from cholesterol in the liver. In CTX, the major bile acid synthesis pathways are disrupted due to partial or total deficiency in sterol 27-hydroxylase encoded by the CYP27A1 gene. CTEXLI may act to replace deficient levels of the…
Pharmacologic Class:
Bile Acid
Indications (1)
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.