5 Key Takeaways

• 1

  The FDA has approved Ctexli (chenodiol) as the first treatment for cerebrotendinous xanthomatosis in adults.

• 2

  Ctexli reduces plasma cholestanol and urine 23S-pentol levels, biomarkers linked to metabolic dysfunction in CTX.

• 3

  CTX is caused by mutations in the CYP27A1 gene, leading to impaired bile acid synthesis and abnormal cholesterol accumulation.

• 4

  The efficacy of Ctexli was established in a 24-week, double-blind, placebo-controlled trial with significant results.

• 5

  Common adverse events of Ctexli include diarrhea, headache, abdominal pain, and liver toxicity monitoring is recommended.