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The efficacy of elafibranor was evaluated in Study 1 (NCT04526665), a multicenter, randomized, double-blind, placebo-controlled study. This study enrolled 161 adult patients with primary biliary cholangitis (PBC) who had an inadequate response to or intolerance of ursodeoxycholic acid (UDCA). Patients were randomly assigned to receive either elafibranor 80 mg (n=108) or placebo (n=53) once daily for a minimum of 52 weeks. When applicable, patients continued their pre-study dose of UDCA throughout the study period.
Patients were enrolled if they had an alkaline phosphatase (ALP) level ≥1.67 times the upper limit of normal (ULN) and a total bilirubin (TB) level ≤2 times the ULN. Patients with other liver diseases or decompensated cirrhosis were excluded.
Mean age: 57 years (range: 36–76 years).
Mean body weight: 70.8 kg (range: 43–134 kg).
Study population: Predominantly female (96%) and White (91%).
Baseline ALP concentration: Mean 321.9 U/L (range: 151–1398 U/L); 39% of patients had a baseline ALP concentration >3 times the ULN.
Baseline TB concentration: Mean 0.56 mg/dL (range: 0.15–1.76 mg/dL); 96% of patients had a baseline TB concentration ≤ULN.
Baseline ALT concentration: Mean 50 U/L (range: 11–188 U/L).
Baseline AST concentration: Mean 46 U/L (range: 14–203 U/L).
Concomitant UDCA use: Most patients (95%) received the study treatment (elafibranor or placebo) in combination with UDCA. Six patients (6%) in the elafibranor group and 2 patients (4%) in the placebo group were intolerant to UDCA and received elafibranor as monotherapy.
Baseline cirrhosis-related indicators: At baseline, 12 patients (11%) in the elafibranor group and 8 patients (15%) in the placebo group met at least one of the following criteria: serum albumin <3.5 g/dL, INR >1.3, TB >1×ULN, liver stiffness measurement (Fibroscan) >16.9 kPa, or a prior biopsy indicating cirrhosis.
FDA，2024.06
