Ipsen’s drug candidate for a rare liver disease secured accelerated approval from the FDA on Monday, making good on a licensing deal with Genfit that dates back to 2021.
The candidate dubbed elafibranor, to be marketed as Iqirvo, was approved for primary biliary cholangitis (PBC). Last week, Jefferies analysts said the drug could reach more than €500 million in peak sales.
Christelle HuguetAhead of the approval announcement, Ipsen’s head of R&D Christelle Huguet told Endpoints News Ipsen is ready to launch elafibranor and “everything is lined up” to that end. Ipsen licensed elafibranor from Genfit in 2021 for $136 million upfront, save for in China, Hong Kong, Taiwan and Macau, where it is licensed to Terns Pharmaceuticals.
PBC is the build-up of bile and toxins in the liver, which is also subject to chronic inflammation. The disease causes scarring of the liver and destruction of bile ducts which worsens over time until patients end up needing a liver transplant.
First-line standard of care ursodeoxycholic acid (UDCA) helps patients manage their disease but does not actually slow down disease progression, Huguet said. And neither does the current second-line treatment option, Ocaliva, according to Huguet, who added that this drug is also linked with worsening of patients’ itch symptoms. “It is well documented that patients continue to progress on these treatments,” Huguet said.
Ocaliva was developed by Intercept Pharmaceuticals and sold to privately-held Italian pharma Alfasigma last year after two FDA rejections in the larger hepatic indication of metabolic dysfunction-associated steatohepatitis, or MASH.
In the Phase 3 ELATIVE trial, just over half of PBC patients who received 80 mg elafibranor achieved the primary endpoint of cholestasis response at 52 weeks versus 4% of placebo patients (p<0.001). Cholestasis response was defined according to alkaline phosphatase and total bilirubin levels, both of which are key biomarkers of liver disease progression.
“Elafibranor is therefore the first option that really has the potential to slow disease progression, and that is something that’s most important for patients,” Huguet said. The drug could be used in combination with first-line options to slow PBC and should help the estimated 40% of patients who do not respond to UDCA at all, she said.
In ELATIVE, the majority of side effects were mild or moderate with no treated patients experiencing severe itch. The most common side effects of elafibranor were gastrointestinal in nature and included abdominal pain, diarrhea, nausea and vomiting.
Elsewhere, Gilead Sciences’ seladelpar for certain second-line PBC patients has an Aug. 14 PDUFA date. The pharma company obtained the asset as part of its $4.3 billion acquisition of CymaBay Therapeutics, which was completed in March.