FDA Grants Accelerated Approval to Ipsen’s Iqirvo® for Primary Biliary Cholangitis

Ipsen has announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Iqirvo® (elafibranor) 80 mg tablets for the treatment of the rare liver disease, primary biliary cholangitis (PBC). This marks the first new medication for PBC in nearly a decade. Approximately 100,000 people in the United States suffer from this condition, which can lead to liver failure if not adequately treated.

The approval is based on the positive results from the Phase III ELATIVE trial. The trial demonstrated that a combination of Iqirvo and ursodeoxycholic acid (UDCA) significantly improved biochemical responses in patients compared to those receiving a placebo. Specifically, the trial revealed that 51% of patients treated with Iqirvo and UDCA achieved a biochemical response, compared to just 4% in the placebo group. Furthermore, secondary endpoints showed that 15% of patients treated with Iqirvo and UDCA achieved normalization in alkaline phosphatase (ALP) levels, a key biochemical marker in PBC.

Christelle Huguet, Executive Vice President and Head of Research and Development at Ipsen, emphasized the importance of this new treatment for PBC patients, many of whom do not respond adequately to existing therapies. Iqirvo offers a much-needed alternative for these patients, providing significant improvements in biochemical markers compared to UDCA alone.

Iqirvo is a first-in-class oral peroxisome proliferator-activated receptor (PPAR) agonist. Ipsen in-licensed the drug from GENFIT in 2021. The approval comes under the FDA's accelerated approval program, which allows for the earlier approval of drugs that treat serious conditions based on a surrogate endpoint. However, continued approval may depend on further verification of clinical benefits in confirmatory trials. Ipsen is currently conducting the ELFIDENCE trial to confirm these benefits.

Dr. Kris Kowdley, Director at Liver Institute Northwest and a primary investigator on the ELATIVE study, stated that Iqirvo provides an effective second-line treatment option for PBC patients, with favorable benefit-risk data. This approval will enable healthcare providers in the U.S. to address the unmet needs of PBC patients more effectively.

Primary biliary cholangitis is an autoimmune liver disease characterized by chronic inflammation and bile duct destruction, leading to irreversible liver fibrosis. It primarily affects women and can significantly impair daily life, causing severe fatigue and debilitating itching.

Carol Roberts, Executive President of PBCers, a patient advocacy organization, highlighted the importance of new treatment options and better education for PBC patients. Many people living with PBC feel misunderstood and uncertain about their disease progression. Early diagnosis and new treatments like Iqirvo are crucial for improving patient outcomes.

Iqirvo has also been submitted to the European Medicines Agency (EMA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA) for approval, with decisions expected in the second half of 2024. The FDA approval of Iqirvo strengthens Ipsen's portfolio in treating rare cholestatic liver diseases. Ipsen already offers FDA-approved treatments for pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS).

Iqirvo is an oral, once-daily medication recommended for adults with PBC who have an inadequate response to UDCA or cannot tolerate it. While the exact mechanism is not fully understood, its pharmacological activity involves inhibiting bile acid synthesis through activation of PPAR-alpha and PPAR-delta.

The ELATIVE study, a multi-center, randomized double-blind, placebo-controlled Phase III clinical trial, evaluated the efficacy and safety of Iqirvo. The trial included 161 participants, most of whom received treatment in combination with UDCA. The study results, published in the New England Journal of Medicine, support the significant biochemical response to Iqirvo.

The most common adverse reactions reported in the study included weight gain, abdominal pain, diarrhea, nausea, and vomiting. To support U.S. patients, Ipsen offers the IPSEN CARES® program, providing educational support and assistance with coverage, access, and reimbursement inquiries.

With this new approval, Ipsen continues to focus on providing transformative medicines in oncology, rare diseases, and neuroscience, leveraging nearly a century of development experience and a global presence.