Ipsen's Iqirvo® (elafibranor) Approved in EU for Primary Biliary Cholangitis

PARIS, France I September 20, 2024 I Ipsen (Euronext: IPN; ADR: IPSEY) announced today that the European Commission has conditionally approved Iqirvo® (elafibranor) 80mg tablets for the treatment of primary biliary cholangitis (PBC). It is approved for use in combination with ursodeoxycholic acid (UDCA) in adults who have not adequately responded to UDCA or as a monotherapy for those unable to tolerate UDCA. Iqirvo is a novel oral medication that functions as a peroxisome proliferator-activated receptor (PPAR) agonist, impacting proteins PPARα and PPARδ, which are crucial for regulating bile acid, inflammation, and fibrosis.

Sandra Silvestri, Chief Medical Officer at Ipsen, expressed excitement over the approval, noting that PBC is a rare liver disease mainly affecting women and has seen little innovation in nearly a decade. Silvestri highlighted that up to half of PBC patients are either intolerant or unresponsive to current treatments. She emphasized that Iqirvo offers a promising new treatment for patients facing disease progression and severe symptoms.

The approval of Iqirvo is based on data from the Phase III ELATIVE trial. This trial showed a significant treatment benefit, with a 47% placebo-adjusted difference (P<0.001) between patients treated with Iqirvo 80mg (51%) and those on placebo (4%) achieving a biochemical response. Although the reduction in PBC Worst Itch-NRS score was greater for those on Iqirvo compared to placebo, it was not statistically significant. The treatment showed an improvement in pruritus (itch) as indicated by lower PBC-40 itch and 5-D itch total scores compared to placebo. Adverse events occurred at similar rates in both the Iqirvo and placebo groups, including serious adverse events and those leading to discontinuation.

Dr. Marco Carbone, Professor of Gastroenterology at the University of Milano-Bicocca and Consultant Hepatologist at the Niguarda Liver Transplant Centre, Milan, welcomed the development. He noted that PBC is a progressive liver disease that can lead to liver failure and necessitate liver transplantation. He highlighted that Iqirvo's potential in managing disease progression and reducing itch is positive for both doctors and patients.

Sindee Weinbaum, a Patient Advocate from the European Liver Patients’ Association, noted that PBC affects individuals differently. Some may experience extreme fatigue or severe itch, while others might show poor biomarker levels, indicating uncontrolled disease. Weinbaum stressed the importance of personalized treatment and the value of having new therapeutic options like Iqirvo for PBC management.

PBC is a rare autoimmune liver disease, affecting predominantly women. It is characterized by the build-up of bile and toxins, leading to chronic inflammation, irreversible fibrosis, and destruction of bile ducts. If untreated, it can result in liver transplant and premature death. Common symptoms include pruritus (itch) and fatigue.

Iqirvo (elafibranor) is an oral medication taken once daily. It activates PPARα and PPARδ, reducing bile toxicity and improving cholestasis by modulating bile acid synthesis, detoxification, and transport. It also has anti-inflammatory effects. The benefits of Iqirvo include reduced alkaline phosphatase and bilirubin levels, which are important indicators of PBC progression and liver function. 

In 2019, the U.S. FDA granted Iqirvo a Breakthrough Therapy Designation for adults with PBC who do not respond adequately to UDCA. The drug received accelerated approval in the U.S. in June 2024 and conditional approval in the EU in September 2024. These approvals are contingent on further verification of clinical benefit. Iqirvo is also under regulatory review by other authorities, including the UK's MHRA. Developed by GENFIT, Ipsen acquired exclusive worldwide rights to elafibranor (excluding China, Hong Kong, Taiwan, and Macau) in 2021.

The ELATIVE trial was a multi-center, randomized, double-blind, placebo-controlled Phase III clinical trial that evaluated the efficacy and safety of elafibranor 80mg daily. The trial involved 161 patients and included a long-term extension study allowing patients to receive elafibranor for up to five years. The study’s primary endpoint was a composite biochemical response, measuring alkaline phosphatase and bilirubin levels at 52 weeks. Full results were published in the New England Journal of Medicine.

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