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Ipsen, Genfit Drug That Missed in MASH Wins FDA Approval in a Much Rarer Liver Disease


A rare liver disease that can progress to organ failure now has a new FDA-approved therapy, a drug from Ipsen that brings a novel approach to treating the chronic condition.

The Ipsen drug, elafibranor, treats primary biliary cholangitis (PBC). The regulatory decision announced late Monday makes the drug just the third approved therapy for the chronic liver disorder, but competition in this indication is heating up. Paris-based Ipsen will market its new product under the brand name Iqirvo.

PBC is an autoimmune condition in which bile and toxins build up in the liver, leading to inflammation and damage to the bile ducts. The lifelong disorder can worsen to the point of requiring a liver transplant. Ipsen estimates that PBC affects 100,000 people in the U.S. Standard treatment is ursodeoxycholic acid (UDCA), an old drug for dissolving gallstones that has an additional approval for treating PBC. UDCA, also called ursodiol, is a naturally occurring bile acid. As a treatment for PBC, this therapy is intended to help bile move through the liver, improving liver function and reducing scarring in the organ.

FDA approval of Iqirvo, a once-daily pill, covers use of the drug in combination with UDCA in adults whose disease responds inadequately to that first-line therapy, or as a monotherapy for those unable to tolerate UDCA. Iqirvo is a small molecule designed to activate peroxisome proliferator-activated receptors (PPAR), a family of receptors that control lipid and glucose metabolism. The exact way that Iqirvo works to treat PBC is not known. But this approach of targeting PPARs was first tried as a treatment for the prevalent fatty liver disease now called metabolic dysfunction-associated steatohepatitis (MASH).

Iqirvo was discovered and developed by Genfit, which advanced the drug into late-stage clinical testing in MASH. In 2020, the Lille, France-based company announced the drug failed to meet the goals of its Phase 3 clinical trial. The company subsequently restructured and focused clinical development of the drug in the much rarer PBC. In 2021, Genfit licensed the drug’s rights to Ipsen for €120 million up front. According to the terms of the deal, Genfit is eligible for up to €360 million in milestone payments as well as royalties from Ipsen’s sales of the new product. Genfit said it expects to receive €89 million in total milestone payments from Ipsen this year, money it will use to finance its liver disease drug R&D.

Iqirvo’s approval sets it up for competition with Ocaliva, a drug from Intercept Pharmaceuticals. Ocaliva’s main ingredient is obeticholic acid, an analog of a bile acid found in humans. The drug binds to a receptor in the liver and intestines that plays a role in inflammation, fibrosis, and metabolism. In 2016, the FDA approved Ocaliva as a second-line PBC treatment. Intercept’s efforts to expand use of the drug to MASH ran into clinical trial and regulatory setbacks. Last year, Intercept was acquired by Italian company Alfasigma, which framed the deal as a way to expand its presence in the U.S. and in liver disease.

More competition is coming. CymaBay Therapeutics drug seladelpar is a small molecule that takes a similar approach as Iqirvo, activating PPAR delta. Seeing seladelpar as a way to expand its presence in liver diseases, Gilead Sciences earlier this year paid $4.3 billion to acquire CymaBay. Seladelpar is currently under FDA review with an Aug. 14 target date for a regulatory decision.

The FDA decision for Iqirvo is an accelerated approval based on data from a placebo-controlled Phase 3 clinical trial. The main goal of this trial was showing a reduction of alkaline phosphatase (ALP), an enzyme whose levels are elevated in PBC patients. Results showed that 51% of patients treated with Iqirvo and UDCA achieved this primary goal. By comparison, 4% of those treated with UDCA and a placebo achieved that response. The most common adverse reactions reported in the study included weight gain, abdominal pain, diarrhea, nausea, and vomiting.

“Data from the pivotal Phase 3 ELATIVE clinical trial demonstrated that Iqirvo is an effective second-line treatment for patients with PBC with favorable benefit and risk data,” Dr. Kris Kowdley, director at Liver Institute Northwest, Washington, and a primary investigator on the study, said in a prepared statement. “The approval of Iqirvo will allow healthcare providers in the U.S. to address an unmet need with the potential to significantly reduce ALP levels for our patients with PBC.”

Iqirvo’s trial results do not show improvement in survival or prevention of liver decompensation. Reducing alkaline phosphatase is a surrogate endpoint, a goal that indicates a drug might be working. The FDA’s accelerated approval decision for the drug may require Ipsen to produce additional clinical data to retain the product’s approval status.

Iqirvo is still under review in Europe, with regulatory decisions expected in the second half of this year. The drug joins a liver disease portfolio that includes Bylvay, a drug that Ipsen added via the acquisition of Albireo last year. Bylvay was initially approved in 2021 as a treatment for pruritus caused by progressive familial intrahepatic cholestasis (PFIC). Nearly a year ago, the drug notched an additional FDA approval for treating pruritus from Alagille syndrome.

Image: Sebastian Kaulitzki/Science Photo Library, Getty Images



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