CTEXLI
Generic: CHENODIOL
Basic Information
Manufacturer
Mirum Pharmaceuticals Inc.
Product Type
HUMAN PRESCRIPTION DRUG
Route of Administration
ORAL
FDA Set ID
015384e7-4ac5-4782-a309-e40cd1421c5b
Indications & Usage
1 INDICATIONS AND USAGE 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 )
CTEXLI is a bile acid indicated for treatment of cerebrotendinous xanthomatosis (CTX) in adults.
( 1 )
Adverse Reactions
6 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.
6.1 Clinical 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.
6.2 Postmarketing 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.
( 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.
6.1 Clinical 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.
6.2 Postmarketing 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.