FDA Approves Ipsen's Primary Biliary Cholangitis Drug

Ipsen's elafibranor, marketed under the name Iqirvo, has secured accelerated approval from the FDA for the treatment of primary biliary cholangitis (PBC) in adults. This marks a significant milestone as it is the first new medication for PBC in almost ten years. The approval follows impressive outcomes from the phase 3 ELATIVE trial, which demonstrated considerable biochemical response improvements over current treatments. In the trial, 70% of patients treated with elafibranor exhibited biochemical improvements, whereas no improvements were observed in the placebo group. These improvements were gauged by reduced levels of alkaline phosphatase and total bilirubin. Additionally, Iqirvo showed benefits in enhancing the quality of life for patients suffering from itch-related symptoms.

PBC is a rare autoimmune liver disease that affects approximately 100,000 individuals in the United States. If left untreated, it can progress to liver failure. Iqirvo offers a new treatment option for patients who do not respond adequately to ursodeoxycholic acid (UDCA) or cannot tolerate it. Approved for use either in combination with UDCA or as a standalone therapy, it provides an alternative for managing this challenging condition.

Iqirvo is a pioneering medication belonging to a new class, designed as an oral, once-daily peroxisome proliferator-activated receptor (PPAR) agonist. By activating PPAR receptors, Iqirvo aids in the regulation of gene expression related to lipid metabolism and inflammation, presenting a novel mechanism of action distinct from existing treatments.

In addition to its FDA approval, Iqirvo is currently under review by the European Medicines Agency (EMA) and the UK Medicines and Healthcare products Regulatory Agency, with decisions anticipated later this year.

Meanwhile, Gilead is also in pursuit of approval for another PBC treatment, seladelpar. Acquired through its recent $4.3 billion purchase of CymaBay Therapeutics, seladelpar is an oral PPAR-delta agonist aimed at liver disease pathways. In its phase 3 trial, 70% of patients responded positively to seladelpar, with 37% achieving normal biomarker levels. The FDA has granted seladelpar priority review status, with an expected decision in August.