Bile Acid

Ctexli

Approved 21 Feb 2025OralTablet

Indications

CTEXLI is indicated for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. CTEXLI is a bile acid indicated for treatment of cerebrotendinous xanthomatosis (CTX) in adults. ( 1 )

Contraindications

None. None. ( 4 )

Warnings and precautions

Hepatotoxicity : Obtain baseline liver transaminase and total bilirubin levels in all patients and monitor yearly and as clinically indicated. Interrupt treatment until the levels have returned to baseline values. For persistent or recurrent liver test abnormalities, consider discontinuing CTEXLI. ( 5.1 )

5.1Hepatotoxicity

Chenodiol, including CTEXLI, has been associated with hepatotoxicity [see Adverse Reactions ( 6 )] . In Trial 1, one CTEXLI-treated patient (7%) had increased ALT levels > 3 times ULN, which led to treatment interruption. Patients with pre-existing liver disease or bile duct abnormalities may be at higher risk for hepatotoxicity during treatment with CTEXLI. Published reports suggest patients who are poor sulfators of lithocholic acid are more likely to develop chenodiol-induced serum aminotransferase elevations [see Clinical Pharmacology ( 12.3 )] .

Obtain baseline liver transaminase (ALT, AST) and total bilirubin levels in all patients prior to treatment initiation with CTEXLI. If liver transaminase levels are elevated > 3 times ULN or total bilirubin level is >2 times ULN, interrupt treatment with CTEXLI until the levels have returned to baseline values. Monitor liver transaminase and total bilirubin levels yearly and as clinically indicated. 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). If clinical signs and symptoms consistent with hepatotoxicity occur, have the patient discontinue CTEXLI immediately.

Interactions

Bile acid sequestering agents and aluminum-based antacids: Avoid concomitant use with CTEXLI. ( 7.1 )
Coumarin and its derivatives: Monitor prothrombin time and adjust dosage accordingly. ( 7.2 )

At a glance

Brand name: Ctexli
Active substance: chenodiol
Pharmacologic class: Bile Acid
Route: Oral
Dosage form: Tablet
Applicant: Mirum
Status: Prescription
FDA approval: 21 Feb 2025
Application: NDA219488-001
RxCUI: 2710417

Warnings and precautions

5.1Hepatotoxicity

Adverse reactions

The following clinically significant adverse reaction is described elsewhere in the labeling:

Hepatotoxicity [see Warnings and Precautions ( 5.1 )] The most common adverse reactions (incidence > 14%) are diarrhea, headache, abdominal pain, constipation, hypertension, muscular weakness, and upper respiratory tract infection. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Mirum Pharmaceuticals at 1-855-MRM-4YOU or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

6.1Clinical Trials Experience

Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety of CTEXLI was evaluated in a randomized, double blind, placebo-controlled, 2-period, 2-treatment crossover trial in 14 patients (16 to 55 years of age) with CTX (Trial 1).

CTEXLI is not approved for use in pediatric patients. The dosage of CTEXLI was 250 mg orally three times a day [see Clinical Studies ( 14 )]. The mean (SD) chenodiol exposure during Trial 1 was 139.1 (26.7) days. The most common adverse reactions which occurred in two or more patients ( > 14%) during CTEXLI treatment (including the two 8-week open-label treatment periods) were diarrhea (36%), headache (21%), and abdominal pain (including abdominal pain upper) (14%), constipation (14%), hypertension (14%), muscular weakness (14%), and upper respiratory tract infection (14%).

In Trial 1, one CTEXLI-treated patient (7%) had increased ALT levels > 3x ULN, which led to treatment interruption.

6.2Postmarketing Experience

The following adverse reactions have been identified during post approval use of chenodiol. Because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure.

Hepatobiliary Disorders: Hepatotoxicity [see Warnings and Precautions ( 5.1 )]
Immune System Disorders: Hypersensitivity reactions such as facial swelling, pruritus, rash, urticaria.

Ctexli

Indications

Contraindications

Warnings and precautions

5.1Hepatotoxicity

Interactions

At a glance

Two medicines in one question?

Ctexli

Indications

Contraindications

Warnings and precautions

5.1Hepatotoxicity

Interactions

At a glance

Two medicines in one question?

7.1Effect of Other Drugs on

7.2Effect of

Pregnancy and breastfeeding

8.1Pregnancy Risk Summary

Adverse reactions

6.1Clinical Trials Experience

6.2Postmarketing Experience

Composition

Official product information