Elafibranor (Iqirvo) is for treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who do not respond adequately to UDCA or as monotherapy in patients unable to tolerate UDCA.
PBC is a rare, chronic cholestatic liver disease that destroys interlobular bile ducts and leads to cholestasis and liver fibrosis.
Left untreated, the disease can worsen over time, leading to cirrhosis and liver transplant and, in some cases, premature death.
PBC also harms quality of life, with patients often experiencing severe fatigue and pruritus.
Elafibranor, an oral dual peroxisome proliferator–activated receptor (PPAR) alpha and delta agonist.
Among 161 patients, a biochemical response was seen in 55 of 108 (51%) who received elafibranor vs 2 of 53 (4%) who received placebo.
At week 52, the ALP level normalized in 15% of patients in the elafibranor group and none of the patients in the placebo group.
Improvement in survival and prevention of liver decompensation events have not been demonstrated and that continued approval for PBC may be contingent upon verification and description of clinical benefit in confirmatory trials.
The most common adverse effects with Iqirvo, reported in ≥ 10% of study participants, were weight gain, abdominal pain, diarrhea, nausea, and vomiting.
Elafibranor is not recommended for people who have or develop decompensated cirrhosis.
Elafibranor is an effective second-line treatment for patients with PBC with favorable benefit and risk data,.