CTEXLI is the first and only FDA-approved treatment for CTX in adults¹

CTEXLI is indicated for the treatment of CTX in adults¹

CTX stems from a mutated CYP27A1 gene, which causes a deficiency in an enzyme (sterol 27-hydroxylase) needed for bile acid synthesis—specifically to make CDCA^1,2
Without sufficient CDCA, negative feedback mechanisms that regulate bile acid synthesis are inhibited^2,3
- This causes an overproduction of toxic bile acid precursors (eg, 23S-pentol, cholestanol)^2,3

CTX takes away CDCA—CTEXLI helps replenish deficient CDCA^1-7

Overproduction of toxic bile acid precursors:
- Cholestanol
- 23S-pentol
Accumulation occurs in tissues (eg, eyes, tendons, brain), which can cause lasting damage or dysfunction
This disruption in bile acid synthesis leads to deficient CDCA levels

CTX mechanism of disease and CTEXLI mechanism of action

CTEXLI helps regulate bile acid homeostasis^1,4

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Significant reductions in toxic bile acid precursors

The RESTORE study assessed the impact of CTEXLI on the overproduction of certain toxic bile acid precursors.¹

See CTEXLI Efficacy Data

References: 1. CTEXLI™ (chenodiol) tablets. Prescribing Information. Mirum Pharmaceuticals, Inc. 2. Freedman SF, Brennand C, Chiang J, et al. Prevalence of cerebrotendinous xanthomatosis among patients diagnosed with acquired juvenile-onset idiopathic bilateral cataracts. JAMA Ophthalmol. 2019;137(11):1312-1316. doi:10.1001/jamaophthalmol.2019.3639 3. Köroğlu M, Karakaplan M, Gündüz E, et al. Cerebrotendinous xanthomatosis patients with late diagnosed in single orthopedic clinic: two novel variants in the CYP27A1 gene. Orphanet J Rare Dis. 2024;19(1):53-65. doi:10.1186/s13023-024-03082-4 4. Badura-Stronka M, Hirschfeld AS, Winczewska-Wiktor A, et al. First case series of Polish patients with cerebrotendinous xanthomatosis and systematic review of cases from the 21st century. Clin Genet. 2022;101(2):190-207. doi:10.1111/cge.14079 5. Fiorucci S, Distrutti E. Chenodeoxycholic acid: an update on its therapeutic applications. Handb Exp Pharmacol. 2019;256:265-282. doi:10.1007/164_2019_226 6. Ribeiro RM, Vasconcelos SC, Lima PLGSB, et al. Pathophysiology and treatment of lipid abnormalities in cerebrotendinous xanthomatosis: an integrative review. Brain Sci. 2023;13(7):1-16. doi:10.3390/brainsci13070979 7. Hoeke MO, Heegsma J, Hoekstra M, Moshage H, Faber KN. Human FXR regulates SHP expression through direct binding to an LRH-1 binding site, independent of an IR-1 and LRH-1. PLOS One. 2014;9(2):e88011. doi:10.1371/journal.pone.0088011

INDICATION

CTEXLI is indicated for the treatment of cerebrotendinous xanthomatosis (CTX) in adults.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Hepatotoxicity: Chenodiol, including CTEXLI, has been associated with hepatotoxicity. Patients with preexisting liver disease or bile duct abnormalities may be at a higher risk for hepatotoxicity during treatment.

Before initiating CTEXLI, obtain baseline liver transaminase (alanine aminotransferase [ALT] and aspartate aminotransferase [AST]) and total bilirubin levels in all patients. Monitor liver transaminase (ALT, AST) and total bilirubin levels yearly and as clinically indicated. If liver transaminase levels are elevated >3 times the upper limit of normal (ULN) or total bilirubin level is >2 times ULN, interrupt treatment until the levels have returned to baseline values. For persistent or recurrent liver test abnormalities, consider discontinuing CTEXLI.

Inform the patient of the symptoms of hepatotoxicity (e.g., abdominal pain, bruising, dark-colored urine, fatigue, bleeding, jaundice, nausea, and pruritus). Have the patient discontinue CTEXLI immediately if clinical signs and symptoms consistent with hepatotoxicity occur.

ADVERSE REACTIONS

The most common adverse reactions (≥14%) during CTEXLI treatment were diarrhea, headache, abdominal pain, constipation, hypertension, muscular weakness, and upper respiratory tract infection.

DRUG INTERACTIONS

Bile acid sequestering agents and aluminum-based antacids: Avoid concomitant use with CTEXLI. Co-administration of bile acid sequestering agents, such as cholestyramine and colestipol, or aluminum-based antacids may decrease absorption of CTEXLI in the intestine and may result in decreased efficacy.

Coumarin and its derivatives: Monitor prothrombin time and adjust the dosage of coumarin or its derivatives if concomitant use with CTEXLI is unavoidable. Due to potential hepatotoxicity, CTEXLI may affect the pharmacodynamics of coumarin and its derivatives, causing unexpected prolongation of the prothrombin time and hemorrhage.

DOSING AND ADMINISTRATION

The recommended dosage of CTEXLI is 250 mg administered orally three times daily, with or without food. Swallow tablets whole.

Please see accompanying full Prescribing Information.

INDICATION

CTEXLI is indicated for the treatment of cerebrotendinous xanthomatosis (CTX) in adults.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

ADVERSE REACTIONS

The most common adverse reactions (≥14%) during CTEXLI treatment were diarrhea, headache, abdominal pain, constipation, hypertension, muscular weakness, and upper respiratory tract infection.

DRUG INTERACTIONS

DOSING AND ADMINISTRATION

The recommended dosage of CTEXLI is 250 mg administered orally three times daily, with or without food. Swallow tablets whole.

Please see accompanying full Prescribing Information.

CTEXLI is the first and only FDA-approved treatment for CTX in adults1

CTX takes away CDCA—CTEXLI helps replenish deficient CDCA1-7

CTEXLI helps regulate bile acid homeostasis1,4

INDICATION

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

ADVERSE REACTIONS

DRUG INTERACTIONS

DOSING AND ADMINISTRATION

INDICATION

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

ADVERSE REACTIONS

DRUG INTERACTIONS

DOSING AND ADMINISTRATION

CTEXLI is the first and only FDA-approved treatment for CTX in adults¹

CTX takes away CDCA—CTEXLI helps replenish deficient CDCA^1-7

CTEXLI helps regulate bile acid homeostasis^1,4