Ipsen's Iqirvo Gains EU Nod for Primary Biliary Cholangitis

The European Commission (EC) has conditionally approved Ipsen's Iqirvo (elafibranor) for treating primary biliary cholangitis (PBC), marking the first new drug for the liver disease in nearly a decade. This approval is for use in combination with ursodeoxycholic acid in adults who don't respond adequately to ursodeoxycholic acid alone. For patients who cannot take ursodeoxycholic acid, Iqirvo can be used as a monotherapy.

This significant decision in Europe follows the U.S. Food and Drug Administration's (FDA) accelerated approval granted in June this year. Both the EC and FDA approvals were based on findings from the Phase III ELATIVE trial (NCT04526665). The trial data revealed that patients receiving 80mg of Iqirvo along with ursodeoxycholic acid experienced greater treatment benefits compared to a placebo group. The results, published in the New England Journal of Medicine, showed that 13 times more patients in the Iqirvo group met the composite primary endpoint of biochemical response.

Iqirvo is an oral peroxisome proliferator-activated receptor (PPAR) agonist. This class of drugs works by activating the proteins PPARα and PPARδ, which are believed to play crucial roles in regulating bile acid, inflammation, and liver fibrosis. PBC is an autoimmune disease that causes a buildup of bile and toxins, leading to inflammation and liver fibrosis. It predominantly affects women, causing symptoms such as joint pain, fatigue, and itchy skin.

Initially, Genfit, the biopharma company that discovered and developed elafibranor, aimed to target the metabolic dysfunction-associated steatohepatitis (MASH) market. However, due to unsatisfactory results from a pivotal Phase III trial, this path was abandoned. Ipsen subsequently acquired worldwide rights to the drug—excluding China, Hong Kong, Taiwan, and Macau—from Genfit for $515 million in 2021. Under this agreement, Genfit will receive a milestone payment of $29.45 million (€26.5 million) following pricing and reimbursement approval in Europe.

Iqirvo is projected to achieve global sales of $361 million by 2030, according to GlobalData’s Pharma Intelligence Centre. This approval in PBC sets the stage for competition with Intercept Pharmaceuticals’ Ocaliva (obeticholic acid), which has been available in the European market since 2016. Like Iqirvo, Ocaliva is approved for use in combination with ursodeoxycholic acid or as a monotherapy for patients unable to take ursodeoxycholic acid.

Intercept Pharmaceuticals has not disclosed its revenue from Ocaliva for the past year, offering only a sales forecast of $310 million to $340 million. Future revenue predictions for Ocaliva are challenging due to its rocky regulatory history. The European Medicines Agency (EMA) reviewed the drug in June and a committee suggested that its clinical benefits did not outweigh its risks, recommending the revocation of its marketing authorization. However, this decision was overturned by the President of the General Court of the European Union, allowing Ocaliva to remain on the market.

Overall, the approval of Iqirvo marks a pivotal development in the treatment of primary biliary cholangitis, providing new hope for patients who have limited treatment options.