FDA Approves Mirum’s Ctexli as First Treatment for Rare Lipid Disorder CTX

The FDA approved Ctexli (chenodiol) as the first treatment for cerebrotendinous xanthomatosis (CTX) in adults123.

CTX is a rare genetic lipid storage disorder caused by mutations in the CYP27A1 gene, affecting cholesterol metabolism13.

Ctexli works by replacing deficient bile acids to facilitate cholesterol metabolism and reduce abnormal metabolite deposits14.

The drug's efficacy was demonstrated in a 24-week clinical trial, showing significant reductions in plasma cholestanol and urine 23S-pentol levels16.

Mirum Pharmaceuticals acquired Ctexli as part of a $210 million deal with Travere Therapeutics in 202327.

The recommended dosage is 250 mg taken orally three times daily146.

Common side effects include diarrhea, headache, abdominal pain, and constipation14.

The prescribing information includes a warning for liver toxicity, particularly in patients with pre-existing liver conditions16.

Ctexli received Priority Review, Fast Track, and Orphan Drug designations from the FDA36.

The approval provides Ctexli with seven additional years of market exclusivity7.

Sources:

1. https://conexiant.com/internal-medicine/articles/fda-approves-first-cerebrotendinous-xanthomatosis-treatment/

2. https://healthexec.com/topics/patient-care/fda-approves-first-therapy-very-rare-lipid-storage-disorder

3. https://www.insideprecisionmedicine.com/topics/precision-medicine/ctexli-is-first-fda-approved-drug-for-rare-metabolic-disease/

4. https://www.drugtopics.com/view/fda-approves-ctexli-for-treatment-of-cerebrotendinous-xanthomatosis

6. https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-cerebrotendinous-xanthomatosis-rare-lipid-storage-disease

7. https://www.fiercepharma.com/pharma/after-210m-asset-purchase-travere-mirum-snags-first-fda-approval-rare-metabolic-disorder