Ipsen has received FDA approval for elafibranor, marketed as Iqirvo, a novel treatment for primary biliary cholangitis (PBC), a chronic liver disease. The announcement marks the third approved therapy for PBC, an autoimmune disorder that causes bile and toxins to accumulate in the liver, leading to inflammation and potential organ failure.
Standard treatment for PBC involves ursodeoxycholic acid (UDCA), which assists bile movement through the liver, improving function and reducing scarring. The newly approved Iqirvo, a once-daily pill, is approved for use alongside UDCA for patients who do not respond adequately to UDCA, or as a standalone treatment for those intolerant to UDCA. Iqirvo activates peroxisome proliferator-activated receptors (PPAR), though its precise mechanism in treating PBC remains unclear.
As reported by Medcity News, the FDA granted Iqirvo accelerated approval based on a Phase 3 trial showing that 51% of patients on Iqirvo and UDCA met the primary goal of reducing alkaline phosphatase (ALP) levels, compared to 4% in the placebo group. Common side effects included weight gain, abdominal pain, diarrhea, nausea, and vomiting.
Dr. Kris Kowdley of Liver Institute Northwest noted that Iqirvo offers an effective second-line treatment option with promising benefit-risk data for PBC patients. Despite the positive trial results, Ipsen may need to provide additional clinical data to maintain FDA approval.