CTEXLI (chenodiol)

In CTX patients, the geometric mean (%CV) maximum plasma concentration (C max ), trough plasma concentration (C trough ), and area under the plasma concentration-time curve (AUC 0-8h ) of chenodiol at steady state following the recommended dosage (250 mg administered orally three times daily) were 3.7 mcg/mL (60%), 0.7 mcg/mL (90%), and 12.5 mcg*h/mL (60%), respectively.

AbsorptionThe median (range) T max of chenodiol following an oral administration in CTX patients was 3 (0.5‑8) hours. Distrib...

The efficacy of CTEXLI for the treatment of patients with CTX was evaluated in Trial 1, which was a randomized, double-blind, placebo controlled, 2-period with 2-treatment crossover trial in patients ≥16 years of age (NCT 04270682). In Trial 1, 14 patients were enrolled and 13 patients were randomized and treated in a crossover withdrawal design to receive either CTEXLI 250 mg or placebo orally three times daily for 4 weeks during 2 double-blind treatment periods. The study also included treatment with CTEXLI 250 mg three times daily during an 8 week run-in period and an 8-week open label peri...

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 Experie...

None. None. ( 4 )

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 ) 5.1 Hepatotoxicity 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 ...

7 DRUG 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 ) 7.1 Effect of Other Drugs on CTEXLI Co-administration of bile acid sequestering agents, such as cholestyramine and colestipol, or aluminum-based antacids may decrease absorption of...