Ipsen primary biliary cholangitis drug gains FDA approval

2024-06-12

临床结果临床3期并购优先审批上市批准

Ipsen's elafibranor, branded Iqirvo, has received accelerated approval from the FDA for treating primary biliary cholangitis (PBC) in adults, marking the first new treatment for PBC in nearly a decade.

The approval is based on positive results from the phase 3 ELATIVE trial, showing significant improvements in biochemical response compared to existing treatments. In the trial, 70% of patients on elafibranor showed biochemical improvement, compared to none on placebo. The improvement was measured by lower alkaline phosphatase levels and total bilirubin. Iqirvo also helped improve itch-related quality of life.

PBC, a rare autoimmune liver disease affecting around 100,000 people in the U.S., can lead to liver failure if untreated. Iqirvo, approved for use in combination with ursodeoxycholic acid (UDCA) or as monotherapy, offers a new option for patients who do not respond adequately to UDCA or cannot tolerate it.

Iqirvo is a first-in-class medication designed as an oral, once-daily peroxisome proliferator-activated receptor (PPAR) agonist. By activating PPAR receptors, Iqirvo helps to regulate gene expression involved in lipid metabolism and inflammation, offering a novel mechanism of action that sets it apart from existing treatments.

The drug is also under review by the EMA and the UK Medicines and Healthcare products Regulatory Agency, with decisions expected later this year.

Gilead is also chasing a PBC approval. Seladelpar, picked up in its recent $4.3 billion acquisition of CymaBay Therapeutics, is an oral PPAR-delta agonist PPAR-delta agonist targeting liver disease pathways. In its phase 3 trial, 70% of patients responded positively to seladelpar, with 37% achieving normal biomarker levels. The treatment is under priority review by the FDA with an anticipated decision in August.

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