Iqirvo Approved for Primary Biliary Cholangitis

On June 10, 2024, the U.S. Food and Drug Administration (FDA) approved Iqirvo^® (elafibranor – Ipsen). It’s a peroxisome proliferator-activated receptor (PPAR) agonist for treating adults with primary biliary cholangitis (PBC). It should be used in combination with ursodeoxycholic acid (UDCA) for those patients who do not fully respond to UDCA. It can also be used as monotherapy for patients who cannot tolerate UDCA. Its recommended dose is 80mg orally once per day. Ipsen plans to launch Iqirvo in the U.S. market within the next week at a wholesale acquisition cost (WAC) of about $11,500 for a month’s supply. Full prescribing information can be found here.

At a Glance

• Brand Drug: Iqirvo (elafibranor)
• Manufacturer: Ipsen and GENFIT
• Date Approved: June 10, 2024
• Indication: for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA
• Dosage Forms Available: 80mg oral tablets
• Launch Date: Within the next week
• Estimated Annual Cost: approximately $138,000 per year
• PBC is an inflammatory autoimmune condition that destroys bile ducts. As a result, bile gradually accumulates in the liver, eventually causing irreversible liver damage. PBC is a major cause of liver transplants.
• PBC affects about 100,000 U.S. patients, but its onset is gradual, so most patients typically are not diagnosed with it until after the age of 30. Between 75% and 90% of patients are women.
• While its mechanism is not fully understood, Iqirvo is believed to function by inhibiting bile acid synthesis through the activation of PPAR-alpha and PPAR-delta receptors.
• Accelerate approval was granted based on the results of the phase III ELATIVE trial. In this trial, a higher percentage of patients receiving Iqirvo showed reduced levels of alkaline phosphatase (ALP), a surrogate marker of response, compared to those receiving standard treatment alone (51% versus 4%, respectively).
• The most frequent adverse events (AEs) were weight gain, abdominal pain, nausea, vomiting, and diarrhea.
• Iqirvo may cause muscle pain (myalgia), or weakness (myopathy), or lead to muscle breakdown (rhabdomyolysis). Patients should be checked for these symptoms before starting the drug, and therapy should be stopped if new or worsening muscle problems occur.
• Liver function tests should also be evaluated before starting treatment with Iqirvo and regularly monitored while receiving therapy.
• Since unborn babies may be injured, women who may become pregnant during treatment with Iqirvo should use reliable forms of contraception (non-hormonal) or add a barrier method of contraception until at least three weeks after therapy ends.
• Ocaliva^® (obeticholic acid – Intercept), a farnesoid X receptor (FXR) agonist, is also indicated for the treatment of PBC in patients whose response to UDCA is considered inadequate (as an add-on to UDCA) and as a monotherapy for patients unable to tolerate UDCA.
• Gilead’s seladeplar is another oral PPAR agonist being developed to treat PBC. The FDA is expected to rule on this drug by Aug. 14, 2024.
• Iqirvo was approved under an Accelerated Approval, so continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. It has also received a Breakthrough Therapy designation.