Safety Profile | CTEXLI™ (chenodiol) | Official HCP Site

Most common adverse reactions (≥14%)¹

Adverse Reaction

Incidence

Diarrhea

36%

Headache

21%

Abdominal pain*

14%

Constipation

14%

Hypertension

14%

Muscular weakness

14%

Upper respiratory tract infection

14%

*Including abdominal pain upper.

The safety and tolerability profile of CTEXLI is based on a mean (SD) CTEXLI exposure of 139.1 (26.7) days in the RESTORE study.¹

Hepatotoxicity¹

Chenodiol, including CTEXLI, has been associated with hepatotoxicity. In the RESTORE study, 1 CTEXLI-treated patient (7%) had increased alanine aminotransferase (ALT) levels >3x upper limit of normal (ULN), which led to treatment interruption. Patients with preexisting 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

Monitoring considerations¹

Obtain baseline liver transaminase (ALT, aspartate transaminase [AST]) and total bilirubin levels in all patients prior to treatment initiation with CTEXLI. If liver transaminase levels are elevated >3x 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 patients of the symptoms of hepatotoxicity (eg, abdominal pain, bruising, dark-colored urine, fatigue, bleeding, jaundice, nausea, and pruritus). If clinical signs and symptoms consistent with hepatotoxicity occur, discontinue CTEXLI immediately

SD=standard deviation.

First and only FDA-approved treatment for CTX in adults

CTEXLI is designed to help reduce the overproduction of toxic bile acid precursors.¹

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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

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.