The European Medicines Agency (EMA) has recommended that a conditional marketing authorization should be granted for Iqirvo (elafibranor; Ipsen Pharma) for treating primary biliary cholangitis (PBC).
PBC is a rare, chronic disease in which the body's immune system mistakenly attacks the interlobular bile ducts in the liver. These ducts become inflamed, and cirrhosis, liver failure, and premature death may occur if not treated.
Symptoms of PBC include itchy skin, fatigue, and bone and joint aches.
Elafibranor is a peroxisome proliferator–activated receptor (PPAR) alpha and delta agonist that works by controlling liver inflammation and the production of bile acids.
The oral treatment is given in combination with ursodeoxycholic acid (UDCA) in adults who have not responded well to UDCA, or as monotherapy in patients unable to tolerate UDCA.
Delayed Development of Liver Fibrosis and Cirrhosis
At its meeting on July 25, the EMA's Committee for Medicinal Products for Human Use agreed that the benefits of Iqirvo are its ability to reduce alkaline phosphatase and bilirubin levels in adults with PBC. The drug's clinical benefits include delayed development of liver fibrosis and cirrhosis, as well as a reduced risk of requiring a liver transplant and early mortality, the committee determined.
A trial, reported in The New England Journal of Medicine in November 2023, found that 51% of 161 patients with PBC who received the drug exhibited significantly greater improvements in biochemical indicators of cholestasis compared with 4% receiving placebo.
The main side effects associated with Iqirvo are abdominal pain, diarrhea, nausea, and vomiting. Iqirvo is not recommended for use in people who have symptoms or signs of advanced liver disease.
Iqirvo will be available as 80-mg film-coated tablets.
Conditional marketing authorizations are given by the EMA to a medicinal product that fulfils an unmet medical need where the benefits of immediate availability are judged to outweigh the risk that additional trial data is needed. The marketing authorization holder is expected to provide such data at a later stage.
The European Commission will have a final say in whether the marketing authorization is granted.