It has been almost a decade since the European medical community welcomed news of an innovation for primary biliary cholangitis (PBC). Therefore, the EC’s recent conditional approval of Ipsen’s drug iqirvo-elafibranori was well received.

Treatment consists of 80mg tablets daily combined with ursodeoxycholic acid (UDCA) in the treatment of PBC patients who are not fully responsive to UDCA. Another option would be monotherapy for adults who were not able to tolerate UDCA.

Iqirvo is a peroxisome proliferator-activated receptor (PPAR) agonist, that has an impact on PPARδ and PPARa proteins, thought to regulate inflammation, bile acid, and fibrosis. Approval of the drug relied on the phase 3 ELATIVE trial which recorded biochemical results of 51% whereas placebo registered 4%.

Patients in both groups reported similar adverse events. However, there were few similarities in the disease itself as every person is affected differently with some people more symptomatic than others.
Mrs. Sindee Weinbaum, Patient Advocate for the European Liver Patients Association, explained that a few people may not have obvious symptoms, yet they may have low biomarker levels which suggests that there is minimal control of the disease.

Mrs. Weinbaum suggested that the doctor and patient take part in constructive discussions about optimum care.

PBC – an Autoimmune and Cholestatic Liver Disorder

PBC is a cholestatic liver disorder which occurs when the flow of bile between the liver and the duodenum accumulates. The accumulation of toxins and bile plus inflammation results in the liver being irreversibly scared and bile ducts being destroyed. The condition may worsen if it is not treated properly resulting in liver transplants or even premature death. PBC is defined as a progressive autoimmune disease-causing inflammation and injury to the small bile ducts found in the liver. PBC is a liver disease classified as rare and found mostly in women. PBC impacts about 100,000 individuals living in the United States.

About Iqirvo

Iqirvo is a peroxisome proliferator-activated receptor (PPAR) agonist, that impacts PPARα and PPARδ. The activation of PPARα and PPARδ decreases bile toxicity. Iqirvo also reduces alkaline phosphatase and bilirubin levels in people with PBC. The conditional approval is contingent upon additional verification of clinical benefit.

About ELATIVE (NCT04526665)

The Phase III ELATIVE trial enrolled 161 patients with PBC randomized to receive 80mg of elafibranor once daily or placebo. The drug was evaluated for safety and efficacy in patients with PBC. ELATIVE was a randomized placebo-controlled Phase III clinical trial. The trial compared the safety and efficacy of elafibranor against placebo for PBC patients who were unable to tolerate UDCA, the standard of care for PBC. All enrolled patients remained with their regimen until they had reported 104 weeks of treatment. The ELATIVE trial is ongoing and allows an additional term of five years to full completion.