Ipsen, Genfit get FDA nod for Iqirvo in liver disorder, setting up Intercept clash

Ipsen's elafibranor, which was licensed from Genfit three years ago, has recently received FDA approval for treating primary biliary cholangitis (PBC), a rare liver disorder. Once considered a possible breakthrough treatment for metabolic-associated steatohepatitis (MASH), elafibranor has now found its niche in addressing PBC, a chronic autoimmune condition affecting around 100,000 people in the U.S., predominantly women aged between 30 and 60.

PBC leads to the accumulation of bile and toxins in the liver, resulting in inflammation and fibrosis. Over time, this condition can lead to severe fatigue and a debilitating itch known as pruritis. Without treatment, PBC can progress to necessitate a liver transplant or even cause early death.

Ipsen plans to charge $11,500 for a month's supply of elafibranor, a first-in-class peroxisome proliferator-activated receptor (PPAR) agonist. Initially explored as a potential treatment for MASH, elafibranor was sidelined due to unsatisfactory data. However, in 2021, Ipsen licensed it from Genfit for 480 million euros ($515 million) to develop it as a PBC treatment.

The FDA's accelerated approval of elafibranor, now branded as Iqirvo, is based on data indicating that an 80 mg tablet can reduce the production rate of the alkaline phosphatase (ALP) enzyme, a crucial biomarker for liver damage. Full approval will hinge on further confirmatory trials proving that Iqirvo can enhance survival rates and prevent complications such as abdominal swelling or gastrointestinal bleeding.

This approval positions Ipsen and Genfit to compete with Intercept Pharmaceuticals’ Ocaliva, which has been available for eight years. Ocaliva was expected to generate sales of between $320 million and $340 million in 2023 before Intercept was acquired by Italian company Alfasigma last year. Analysts predict that Iqirvo could achieve sales of 193 million euros ($207 million) by 2027, with peak sales potentially reaching 400 million euros ($429 million).

Alfasigma completed its acquisition of Intercept shortly after the FDA rejected Ocaliva's application for approval in MASH for the second time in three years. Both Iqirvo and Ocaliva are approved for use in combination with ursodeoxycholic acid (UDCA) or as standalone treatments for patients unable to take UDCA.

Analysts from ODDO BHF view Ipsen's approval of Iqirvo as a significant success, reflecting the active business development strategy under CEO David Loew, who took over in 2020. This approval also raises expectations for future partnerships. However, analysts caution that Iqirvo's approval did not include a reduction in pruritis, a notable drawback when compared to potential competitor CymaBay Therapeutics’ seladelpar, which is anticipated to receive approval for the same indication in August.

Interestingly, Gilead acquired CymaBay earlier this year in a $4.3 billion deal, gaining access to seladelpar. This move underscores the competitive landscape in the PBC treatment market, with multiple companies vying to offer the most effective solutions for this challenging liver disorder.