RegulationPriority Review (United States), Breakthrough Therapy (United States), Fast Track (United States), Accelerated Approval (United States), Orphan Drug (United States), Orphan Drug (European Union), Priority Review (China), Orphan Drug (Japan), Orphan Drug (South Korea)

Structure/Sequence

Molecular FormulaC22H24O4S

InChIKeyAFLFKFHDSCQHOL-UHFFFAOYSA-N

CAS Registry824932-88-9

Clinical Trials associated with Elafibranor

NCT07387549

/ RecruitingPhase 3

A Phase III, Multicentre, Randomised, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Elafibranor in Adult Participants With Primary Sclerosing Cholangitis

The purpose of this study is to find out how well and safely elafibranor works compared to placebo in adult participants with Primary sclerosing cholangitis (PSC). PSC is a rare disease that causes inflammation and scarring of the bile ducts in the liver. Over time, this can lead to liver damage and serious health problems, including the need for a liver transplant and death.
In this study, about 350 participants with large duct PSC will take part. Participants will be randomized to receive either elafibranor 120 mg once daily or a placebo (a tablet with no active medicine). The study includes a screening period, an treatment period, and a post-treatment safety follow-up.
During the study, participants will undergo routine clinical assessments, laboratory testing, imaging evaluations, and complete patient-reassessments to evaluate liver disease progression, symptoms, quality of life and safety.
Following the end of treatment, participants will complete a safety follow-up period at approximately four weeks. Participants may withdraw from the study at any time. Each participant may be in the study for several years, as the treatment period will continue until the study reaches enough health events among participants, which is expected to take about 5 years.

Start Date14 May 2026

Sponsor / Collaborator

Ipsen SA

DRKS00039209

/ RecruitingNot Applicable

Safety and efficacy of elafibranor therapy in patients with primary biliary cholangitis and liver cirrhosis. - Elafibranor in patients with primary biliary cholangitis and liver cirrhosis.

Start Date16 Mar 2026

Sponsor / Collaborator-

NCT06730061

/ Active, not recruitingPhase 3

A Phase III, Open-label, Single Arm Study to Investigate the Efficacy and Safety of Elafibranor 80 mg in Adult Japanese Participants With Primary Biliary Cholangitis (PBC)

The purpose of this study is to find out about the safety and how well the study intervention (elafibranor) works in participants with PBC. The participants in this study will have confirmed PBC with inadequate response or intolerance to UDCA, which is a medication used in the management and treatment of cholestatic liver disease.
PBC is a slowly progressive disease characterised by damage of the bile ducts in the liver, leading to a build-up of bile acids which causes further damage. The liver damage in PBC may lead to scarring (cirrhosis).
PBC may also be associated with multiple symptoms. Many patients with PBC may require a liver transplant or may die if the disease progresses and a liver transplant is not done. In this study all participants will receive a daily dose of elafibranor (the study intervention).
The main aim of this study is to determine if elafibranor reduces alkaline phosphatase (ALP) and total bilirubin levels. High ALP and bilirubin levels in the blood can indicate liver disease.
There will be 4 periods in this study: A screening period (up to 10 weeks) to assess whether the participant can take part. A treatment period (52 weeks) where all eligible participants will receive elafibranor. A variable treatment extension period (2-5 years) from End Of Treatment (EOT) period up to the commercial availability of elafibranor in Japan. A follow-up period (4 weeks) where participants' health will be monitored.
Participants will undergo blood sampling, urine collections, physical examinations, clinical evaluations, electrocardiograms (ECG: recording of the electrical activity of heart), ultrasound examinations (a non-invasive test that passes a probe over skin to look at the bladder, urinary tract, and liver), and Fibroscan® examinations (to measure stiffness of the liver). They will also be asked to fill in questionnaires. Each participant will be in this study for up to approximately 6 years

Start Date16 Jan 2025

Sponsor / Collaborator

Ipsen SA

100 Clinical Results associated with Elafibranor

100 Translational Medicine associated with Elafibranor

100 Patents (Medical) associated with Elafibranor

165

Literatures (Medical) associated with Elafibranor

01 Aug 2026·European Journal of Internal Medicine

From unmet needs to new possibilities: PPAR-targeted therapies in the journey of people living with Primary Biliary Cholangitis

Review

Author: Meschisi, Pietro ; Buzias, Maria ; Calabrese, Maria Pia ; Ronca, Vincenzo ; Ruiu, Antonella ; Plebani, Riccardo ; Lleo, Ana

Primary biliary cholangitis (PBC) is a chronic cholestatic liver disease characterised by progressive immune-mediated destruction of the small intrahepatic bile ducts, leading to cholestasis, fibrosis, and ultimately cirrhosis. Despite therapeutic advances, up to 40% of patients fail to achieve an adequate biochemical response to first-line ursodeoxycholic acid (UDCA) and require second-line therapy. This narrative review traces the patient-physician journey from initial clinical suspicion through diagnosis, risk stratification, treatment selection, and long-term comorbidity management, with particular emphasis on the emerging role of peroxisome proliferator-activated receptor (PPAR) agonists. Fibrates are PPAR agonists that have been used for long time in PBC with evidence of their effect to improve biochemical response in PBC; however, their use remains off-label and they have undergone only limited safety and efficacy evaluation. Novel PPAR agonists-including seladelpar (PPARδ) and elafibranor (PPARα/δ)-have recently received conditional approval following phase 3 trials demonstrating significant reductions in alkaline phosphatase (ALP), alongside clinically meaningful improvements in pruritus and fatigue. These agents offer a favourable benefit-risk profile and represent an important therapeutic advance. In conclusion, effective management of PBC requires a holistic strategy integrating early diagnosis, individualised risk stratification, and timely escalation of targeted therapy. PPAR agonists mark a significant evolution in treatment by addressing biochemical disease activity, symptom burden, and quality of life simultaneously. Systematic identification and management of autoimmune and metabolic comorbidities remain essential throughout the patient journey.

01 Jul 2026·Gastroenterology & hepatology

A Review of Therapies for Primary Biliary Cholangitis.

Article

Author: Kowdley, Kris V ; Alamgir, Mariam ; Bhushan, Sheena ; Sohal, Aalam

Primary biliary cholangitis (PBC) is a rare, chronic, progressive autoimmune cholestatic liver disease that can lead to cirrhosis if left untreated. Ursodeoxycholic acid remains the standard first-line therapy; however, approximately 40% of patients exhibit an inadequate or partial biochemical response, and approximately 5% to 10% of patients are intolerant to the drug. Consequently, there remains a substantial need for effective second-line agents. Until recently, obeticholic acid (OCA) and fibrates were the only available second-line therapies. However, the therapeutic landscape for PBC has evolved significantly with the US Food and Drug Administration conditional approval of elafibranor and seladelpar as second-line therapies for patients without cirrhosis. Notably, OCA has been withdrawn as an available treatment option, further shifting the current treatment paradigm. This article aims to examine the current pharmacologic landscape of first-line and second-line PBC treatments, with an emphasis on clinical considerations and strategies for individualized treatment selection to optimize patient outcomes.

01 Jun 2026·CURRENT THERAPEUTIC RESEARCH-CLINICAL AND EXPERIMENTAL

Understanding Pharmacokinetic-Drug Interactions With Drugs Approved by the US Food and Drug Administration in 2024 to Better Manage the Risk of Drug Interactions With Concomitant Medications: A Review of Clinical Data From New Drug Applications

Review

Author: Owens, Katie ; Argon, Sophie ; Yu, Jingjing ; Ragueneau-Majlessi, Isabelle ; Wang, Yan

Objective:

This analysis aimed to provide a mechanistic understanding and an evaluation of the clinical relevance of pharmacokinetic drug-drug interactions (DDIs) associated with drugs approved by the Food and Drug Administration in 2024.

Methods:

Drug metabolism, transport, and absorption-based DDI data available in New Drug Applications (NDAs) of small molecular drugs approved (n = 34) was analyzed. The mechanism and clinical outcome of these interactions were characterized based on in vitro, in silico, and clinical data.

Results:

As objects, 11 drugs were identified as clinical substrates. Of these, 7 drugs were substrates of CYP3A, 3 of CYP2C9, one of CYP1A2, and one of CYP2C8, including the sensitive substrates vanzacaftor (CYP3A) and vorasidenib (CYP1A2). As precipitants, 6 drugs (acoramidis, cefepime/enmetazobactam, givinostat, lazertinib, mavorixafor, and resmetirom) were clinical inhibitors of CYP enzymes (2C8, 2C9, 2D6, 2E1, and 3A), with mavorixafor being a CYP2D6 strong inhibitor. Two drugs (elafibranor and tovorafenib) showed weak induction of CYP3A. Regarding transporter data, 3 drugs were substrates of transporters, including seladelpar (BCRP and OAT3), sulopenem (OAT3), and vadadustat (OAT1/3), and 8 drugs (arimoclomol, danicopan, givinostat, lazertinib, mavorixafor, resmetirom, vadadustat, and vazacaftor/tezacaftor/deutivacaftor) were inhibitors of transporters. All clinical DDIs with AUC changes ≥ 2-fold triggered labeling recommendations. Several DDIs with an AUC change <2 also had labeling recommendations, pertaining most often to the concomitant use of drugs with a narrow therapeutic index.

Conclusions:

Mechanistic DDI insights from newly approved therapies can be extrapolated to inform the management of commonly co-administered drugs, supporting a safer and more effective use of new drug products in the context of polypharmacy.

243

News (Medical) associated with Elafibranor

03 Sep 2026

·www.biospace.com

GENFIT to Host Virtual KOL Event Focused on MASH Diagnostics on September 9, 2026

Presentations will highlight the clinical utility and long-term commercial opportunity of GENFIT’s proprietary NIS platform, a non-invasive diagnostic technology for MASH Lille (France), Cambridge (Massachusetts, United States), Zurich (Switzerland), September 3, 2026 - GENFIT (Euronext: GNFT) , a biopharmaceutical company dedicated to improving the lives of patients with rare and life-threatening liver diseases, today announced that it will host a virtual key opinion leader (KOL) event on Wednesday, September 9, 2026 at 10:00 AM ET. The event will highlight the evolving landscape of diagnostic testing for Metabolic dysfunction-Associated SteatoHepatitis (MASH) and will feature Dr. Vlad Ratziu, MD, PhD (Sorbonne University, Pitié-Salpêtrière Hospital, Institute for Cardiometabolism and Nutrition, Paris). To register, click here . Professor Ratziu will discuss the clinical unmet need in MASH diagnostics and highlight the clinical utility of GENFIT’s non-invasive testing technology, NIS4® and new generation NIS2+®, at a time when treatment options are expanding rapidly. GENFIT’s technology is recognized by international expert consortia as a leading non-invasive solution which is uniquely positioned, as it captures both disease activity and fibrotic burden, helping identify at-risk MASH patients and supporting more informed clinical decision-making. The technology is included in both European and U.S. clinical guidelines. In a recent milestone for the field, Labcorp's NASHnext®, based on NIS4®, became eligible for reimbursement under the Clinical Laboratory Fee Schedule for patients who meet coverage criteria, effective August 10, 2026. This development may support broader adoption and pave the way for future coverage by private insurers. Company management will then provide perspectives on the commercial opportunity, revenue potential, and GENFIT's development strategy aimed at further broadening patient access. ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); END ABOUT VLAD RATZIU, MD, PHD Professor Vlad Ratziu received his medical training at Paris V, René Descartes University, completed a two-year research fellowship at the Liver Center at UCSF, and earned a doctoral degree from Paris VII Diderot University for his work on the pathophysiology of viral and metabolic liver fibrosis. His research centers on steatotic liver disease; the mechanisms, risk factors, and progression of liver fibrosis in viral and metabolic disease; and the treatment of viral hepatitis and hepatocellular carcinoma. He has coordinated or participated in numerous therapeutic trials in MASH and in European FP7, Horizon 2020, and Innovative Medicines Initiative 2 consortia. He is founder and organizing-committee member of the NASH–TAG meetings. He has published more than 460 articles (H-index of 113) in top-tier specialty journals and has been named among Clarivate's top 1% Highly Cited Researchers from 2022 to 2025. ABOUT NASHNEXT®, NIS4® AND NIS2+® ezstandalone.cmd.push(function () { ezstandalone.showAds(132); }); With a high and growing prevalence of metabolic risk factors in the U.S., early detection of at-risk MASH could benefit a U.S. patient population in the high tens of millions of individuals, highlighting a significant unmet medical need. Liver biopsy currently remains the clinical standard for diagnosing at-risk MASH, but its use is highly constrained by its invasive and costly nature and difficult scalability. Labcorp’s NASHnext® is a non-invasive diagnostic test to easily identify patients with at-risk MASH (Metabolic dysfunction-Associated SteatoHepatitis). It is powered by GENFIT’s proprietary non-invasive diagnostic technology NIS4®. NIS4® and next-generation technology NIS2+® have been widely recognized over the past few years as showing a unique performance in identifying patients with at-risk MASH. This recognition is reflected through a growing body of scientific evidence (including publications in The Lancet and Nature Medicine ), inclusion in joint guidelines from several international scientific societies (EASL-EASD-EASO), strong visibility within major consortia in the U.S. and Europe (NIMBLE and LITMUS), and its use in most large clinical trials conducted by key industry players in MASH. Today NASHnext® is available through Labcorp OnDemand, a digital interface where patients can order the test online. GENFIT is actively seeking to advance partnerships with pharmaceutical companies to support wider deployment of non-invasive testing for at-risk MASH and accelerate adoption in line with the expansion of MASH therapies. GENFIT is also exploring the development of an IVD-labeled (In Vitro Diagnostic) version of its non-invasive diagnostic technology, either independently or in collaboration with a diagnostic-focused partner, with the selected approach expected to reflect the most appropriate route to maximize long-term value for GENFIT. ABOUT GENFIT GENFIT is a biopharmaceutical company committed to improving the lives of patients with rare, life-threatening liver diseases whose medical needs remain largely unmet. GENFIT is a pioneer in liver disease research and development with a rich history and a solid scientific heritage spanning more than two decades. Today, GENFIT focuses on Acute on-chronic Liver Failure (ACLF) and associated conditions such as acute decompensation (AD) and hepatic encephalopathy (HE). It develops therapeutic assets which have complementary mechanisms of action, selected to address key pathophysiological pathways. GENFIT also targets other serious diseases, such as cholangiocarcinoma (CCA), urea cycle disorders (UCD) and organic acidemia (OA). Its R&D portfolio, covering several stages of development, ensures a constant news flow. GENFIT's expertise in developing high-potential molecules – from early to advanced pre-commercialization stages – culminated in 2024 with the accelerated approval of Iqirvo® (elafibranor) by the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA) in the United Kingdom for second-line treatment of Primary Biliary Cholangitis (PBC). Iqirvo® is now marketed in several countries. 1 Beyond therapies, GENFIT also has a diagnostic franchise including NIS2+® for the detection of Metabolic dysfunction-associated steatohepatitis (MASH, formerly known as NASH for non-alcoholic steatohepatitis). GENFIT, a BCorp™ certified company since 2025, is headquartered in Lille, France and has offices in Paris (France), Zurich (Switzerland) and Cambridge, MA (USA). The Company is listed on the Euronext regulated market in Paris, Compartment B (Euronext: GNFT). In 2021, Ipsen became one of GENFIT's largest shareholders, acquiring an 8% stake in the Company's capital. FORWARD LOOKING STATEMENTS ezstandalone.cmd.push(function () { ezstandalone.showAds(133); }); This press release contains certain forward-looking statements with respect to GENFIT, including, but not limited to, statements regarding the clinical utility, commercial potential, adoption and future development of GENFIT's proprietary non-invasive diagnostic technologies, including NIS4® and NIS2+®, the potential impact of reimbursement and payer coverage on adoption of such technologies, the market opportunity and revenue potential associated with GENFIT's diagnostic franchise, and GENFIT's in vitro diagnostic (IVD) development strategy and objectives to broaden patient access. The use of certain words, such as "believe", "potential", "expect", “target”, “may”, “will”, "should", "could", "if" and similar expressions, is intended to identify forward-looking statements. Although the Company believes its expectations are based on the current expectations and reasonable assumptions of the Company’s management, these forward-looking statements are subject to numerous known and unknown risks and uncertainties, which could cause actual results to differ materially from those expressed in, or implied or projected by, the forward-looking statements. These risks and uncertainties include, among others, the uncertainties inherent in research and development, including in relation to non-clinical and pre-clinical programs, reproducibility of preclinical results, the translation of animal model data to human biology, in relation to safety of drug candidates, cost of, progression of, and results from, our ongoing and planned clinical trials, patient recruitment, review and approvals by regulatory authorities in the United States, Europe and worldwide, of our drug and diagnostic candidates, pricing, approval and commercial success of elafibranor in the relevant jurisdictions, exchange rate fluctuations, and our continued ability to raise capital to fund our development, as well as those risks and uncertainties discussed or identified in the Company’s public filings with the AMF, including those listed in Chapter 2 "Risk Factors and Internal Control" of the Company's 2025 Universal Registration Document filed on April 3, 2026 (no. 26-0221) with the Autorité des marchés financiers ("AMF"), which is available on GENFIT's website ( ) and the AMF's website ( ), and those discussed in reports filed with the AMF or otherwise made public, by the Company. In addition, even if the results, performance, financial position and liquidity of the Company and the development of the industry in which it operates are consistent with such forward-looking statements, they may not be predictive of results or developments in future periods. These forward-looking statements speak only as of the date of publication of this press release. Other than as required by applicable law, the Company does not undertake any obligation to update or revise any forward-looking information or statements, whether as a result of new information, future events or otherwise. CONTACTS GENFIT | Investors Jean-Christophe Marcoux – Chief Corporate Affairs Officer | Tel: +33 3 2016 4000 | jean-christophe.marcoux@genfit.com GENFIT | Media Bruno ARABIAN – Agence Maarc | Tel : 06 87 88 47 26 | bruno.arabian@maarc.fr ezstandalone.cmd.push(function () { ezstandalone.showAds(134); }); Stephanie Boyer – Press relations | Tel: +333 2016 4000 | stephanie.boyer@genfit.com GENFIT | 885 Avenue Eugène Avinée, 59120 Loos - FRANCE | +333 2016 4000 | 1 Elafibranor is marketed and commercialized, notably in the U.S and Europe, by Ipsen under the trademark Iqirvo® Attachment ezstandalone.cmd.push(function () { ezstandalone.showAds(135); }); GENFIT to Host Virtual KOL Event Focused on MASH Diagnostics on September 9, 2026

20 Aug 2026

·www.biospace.com

Regeneron wins FDA approval to challenge Ipsen for rare bone disease market

iStock, nambitomo Regeneron’s Pasatru is the second fibrodysplasia ossificans progressiva drug approved in the U.S., following the 2023 approval of Ipsen’s Sohonos. Regeneron has won FDA approval for garetosmab in fibrodysplasia ossificans progressiva, giving the biotech a commercial product built on its scientific breakthrough in the ultrarare disease. It also sets up a showdown with Ipsen, which owns the only other FOP drug on the market. The Thursday approval covers the use of Pasatru—an antibody called garetosmab and REGN2477 during development—to reduce the formation of new heterotopic ossification (HO) lesions and clinician-assessed flare-ups in adults with FOP. HO is characterized by bone developing in soft tissues. Pasatru is the second FOP drug to secure U.S. approval. The first, Ipsen’s Sohonos, came to market in 2023 after a troubled development program marked by clinical and regulatory setbacks. Ipsen acquired the asset in 2019 from Clementia Pharmaceuticals. For the first half of 2026, Sohonos contributed to the €11.4 million ($13.4 million) in sales generated by Ipsen’s rare disease drugs apart from Iqirvo and Bylvay. Following Clinical Hold and Trial Setback, Ipsen Takes $728 Million Hit on FOP Drug Ipsen’s decision to pause dosing in its studies of palovarotene trials for patients with fibrodysplasia ossificans progressive (FOP) has hit the France-based company right in the pocketbook. February 13, 2020 · 2 min read · Alex Keown Read more Regeneron has now won approval based on Phase 3 trial data that showed two doses of Pasatru reduced the total number of new HO lesions compared to placebo. Investigators saw 19 new lesions in the 21 patients on placebo, compared to three new lesions in the 42 patients treated across two Pasatru dose cohorts. The recommended starting dosage is 10 mg/kg, the higher of the two doses given in the Phase 3 trial. Patients receive the antibody intravenously for one hour every month. If a patient doesn’t tolerate the dose, they can drop down to the lower Phase 3 dose, 3 mg/kg. Patients on the lower dose still receive a 60-minute infusion once monthly. Regeneron designed Pasatru to be given in a range of care settings, including at home when appropriate. The flexibility reflects the significant mobility challenges faced by many people with FOP, which can make walking difficult as bone builds up in muscles, tendons, ligaments and other connective tissues. Most patients are wheelchair-bound by age 30, Regeneron said. Regeneron’s efforts in the disease can be traced back to 2015, when researchers published a paper describing an unexpected discovery about the driver of HO in FOP. By looking at FOP in mice, Regeneron identified Activin A as the ligand responsible for HO. Regeneron started a Phase 1 trial of the anti-Activin A antibody—now known as Pasatru— in 2016. About 900 people are diagnosed with FOP. Rare disease–focused biopharma Mirum Pharmaceuticals has predicted that its rival FOP drug candidate, zilurgisertib, could generate peak sales of more than $200 million. Zilurgisertib, which Mirum licensed from Incyte, is under review at the FDA and has an approval decision date of Sept. 26. Mirum is seeking approval of zilurgisertib for the treatment of FOP in patients 12 years of age and older. Regeneron’s Phase 3 study only enrolled adults, although the company plans to start a trial in children and adolescents this year.

Drug ApprovalPhase 3Phase 1AcquisitionClinical Result

31 Jul 2026

·www.fiercebiotech.com

‘It’s making a difference’: Madrigal CEO talks strategy behind MASH shopping spree

Not content with navigating Rezdiffra’s trajectory to blockbuster territory, Madrigal Pharmaceuticals has spent 2026 shopping for a range of drugs that the company hopes could work alongside its metabolic dysfunction-associated steatohepatitis (MASH) trailblazer. Madrigal kicked off the year paying $50 million to Pfizer for the rights to a DGAT2i inhibitor, before spending $60 million the following month on six preclinical siRNA programs from China’s Ribo Life Science and its Swedish subsidiary Ribocure Pharmaceuticals that Madrigal wants to use to silence genes implicated in MASH. The company explained at the time that both deals were aimed at securing therapies that could be combined with Rezdiffra. The biopharma was back at the dealmaking table in May, paying $25 million upfront to Arrowhead Pharmaceuticals for a phase 1-stage RNA interference therapeutic that had previously been rejected by Johnson & Johnson. Early clinical data had suggested the asset could reduce liver fat in a mutation found in 30% of MASH patients, including high prevalence in Hispanic populations. Combined with a preclinical GLP-1 agonist licensed from China’s CSPC Pharmaceutical for $120 million upfront last year, Madrigal now has a diverse array of MASH assets coming up behind Rezdiffra. So what’s the strategy behind this rapid pipeline expansion? “The question early on was what mechanisms we wanted,” Madrigal CEO Bill Sibold told Fierce in an interview at the company’s office in Waltham, Massachusetts. “My answer was: we want all of them,” Sibold continued. “We want anything that makes sense mechanistically or has proven already to have an effect on MASH.” It’s a sign of just how much Rezdiffra has changed the game since being approved as the first-ever treatment for MASH in 2024. Before that point, Sibold said he had counted 23 drugs across the industry that had failed to treat the fatty liver disease previously known as nonalcoholic steatohepatitis (NASH). This rollcall of failure meant MASH had become “the graveyard of drug development,” lumped in with other difficult-to-treat conditions like sepsis and lupus, he remembered. The disease occurs when fat builds up in the liver, causing inflammation and eventually cell death. It is the leading cause of liver transplants for women and the second biggest cause for men in the U.S. “Industry had been really interested in this area for a long time, but nobody could crack the nut,” Sibold said. Many companies tried and failed to find a breakthrough. Gilead’s ASK1 inhibitor selonsertib and its LOXL2 enzyme-targeting simtuzumab both flopped in the clinic, as did Genfit’s dual PPAR alpha/delta agonist elafibranor and Conatus Pharmaceuticals' pan-caspase inhibitor emricasan. Rezdiffra’s origins go back to Roche. Madrigal founder Rebecca Taub, Ph.D., left the Swiss pharma’s metabolic unit to work for VIA Pharmaceuticals, which licensed the thyroid hormone receptor (THR)-β agonist. Madrigal assumed the rights to that agreement when it was founded in 2011 and continued to develop the therapy. Sibold remembers getting a call from a recruiter about the Madrigal CEO position in 2023 while he was president for North America at Sanofi, having helped launch immunology blockbuster Dupixent. “There's no way I'm going to leave this to go to that,” he remembers of his initial reaction to the offer. But as Sibold dug into the company, educating himself on the trials and the science behind its lead asset, he became convinced that this was a unique opportunity to make a special impact. The year after Sibold joined up, Madrigal had secured Rezdiffra’s historic FDA approval. As well as the company's headquarters in West Conshohocken, Pennsylvania, the company now also has an office in Massachusetts—which Fierce visited—as well as an international outpost in Switzerland. “There were so many Big Pharma companies trying to solve [MASH], and this little Pennsylvania company of very few people had the tenacity, the guts and the grit to get there,” Sibold said. Madrigal reported $958.4 million in annual net sales for the drug in 2025. By the second quarter of this year, Rezdiffra was bringing in $364.3 million, equivalent to year-over-year growth of 71%. Sibold told Fierce that it wasn’t always this way. Early on, a MASH advocate told him: “We didn't know whether to believe you at first because it seemed like such a huge hill to climb that you guys didn't even have an approved product. But you are doing exactly what you said you would do.” After establishing itself as the market leader in MASH, what’s next? Does the company stay in its lane, expand into immunology targets or look at new indications? For Sibold, the answer lies in Madrigal’s five-word purpose: leading the fight against MASH. The biopharma wants to continue working with the MASH community it has come to know so well to build out its pipeline with more effective and comprehensive treatments for different subpopulations. Rather than generate data and hope to get bought by a bigger fish, Sibold said he is planning for an independent and sustainable company 10 to 20 years down the line. His vision is for MASH treatments to grow like other specialty markets, such as rheumatoid arthritis, psoriasis or inflammatory bowel disease, with dozens of products that generate tens of billions of dollars in annual sales. “We feel like MASH will emerge like that, and as a result, we want to have the portfolio of products that can best help all or subpopulations of patients,” Sibold said. Unlike a Big Pharma, where MASH may be one of a myriad of indications being focused on, Sibold suggested Madrigal's strength comes from the fact it is committed to the disease. This approach is reflected in trial enrollment and feedback, he said. “The community values expertise, and I think we've made a conscious decision to be experts in MASH,” he said. “Through expertise comes insight into what might be the next best mechanism, or the next best mechanism to combine with your current mechanism, or the best way to design the next trial.” Because reducing liver fat is so important to preventing or treating MASH, GLP-1s are moving into the space and posing a potential threat. Last year, Novo Nordisk’s Wegovy was approved to treat MASH, and some people have warned Sibold that Madrigal could be crushed by GLP-1s in the future. Instead, the CEO insisted more activity in the space is a good thing for Madrigal. “It's great to have another company who's putting resources into diagnosis, awareness and treatment,” he said. “There are some patients that are getting Wegovy, but it is helping to grow the market. It's not to our detriment.” But other Big Pharmas are circling. Last year saw GSK acquire the FGF21 analog efimosfermin from Boston Pharmaceuticals for $1.2 billion upfront, while Roche picked up a similar drug called pegozafermin as part of its $3.5 billion buyout of 89bio . Madrigal may be looking to become the MASH version of what Vertex Pharmaceuticals has achieved in cystic fibrosis, but that doesn’t mean Sibold and his team couldn’t eventually broaden their focus. “Would we look at other diseases? Yes. We would in time, or if the biology takes us there,” he told Fierce. But for now, the company is leaning into MASH specialization, developing relationships with patient groups, clinicians and researchers while raising Rezdiffra’s profile and growing a specialized portfolio of complementary therapies. “It's been fun. It's making a difference. It's helping patients,” Sibold said. “This is as good as it gets.

AcquisitionPhase 1Drug Approval

100 Deals associated with Elafibranor

External Link

KEGG	Wiki	ATC	Drug Bank
D11208	Elafibranor	-	DB05187

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Primary Biliary Cholangitis	United States	Ipsen Biopharmaceuticals, Inc.	10 Jun 2024

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Cholangitis, Sclerosing	Phase 3	United States	Ipsen SA	14 May 2026
Fibrosis	Phase 3	United States	Genfit SA	01 Mar 2016
Fibrosis	Phase 3	Argentina	Genfit SA	01 Mar 2016
Fibrosis	Phase 3	Australia	Genfit SA	01 Mar 2016
Fibrosis	Phase 3	Belgium	Genfit SA	01 Mar 2016
Fibrosis	Phase 3	Canada	Genfit SA	01 Mar 2016
Fibrosis	Phase 3	Chile	Genfit SA	01 Mar 2016
Fibrosis	Phase 3	Colombia	Genfit SA	01 Mar 2016
Fibrosis	Phase 3	Czechia	Genfit SA	01 Mar 2016
Fibrosis	Phase 3	Denmark	Genfit SA	01 Mar 2016

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04526665 (ELATIVE, EASL2026)	Phase 3	Primary Biliary Cholangitis	159	Elafibranor 80 mg	jwegypqmni(ztozwqizhf) = khtrybcoaq lgdcwmykvq (clvainxluk ) View more	Positive	27 May 2026
				Placebo	jwegypqmni(ztozwqizhf) = ljbnssxnlr lgdcwmykvq (clvainxluk ) View more
EASL2026	Not Applicable	Primary Biliary Cholangitis	80	Elafibranor 80 mg/day (Observation)	awfireattz(vbevvflhqp) = zlhvupczlt gjdslfebyt (aypubdxniq ) View more	Positive	27 May 2026
NCT04526665 (ELATIVE, EASL2026)	Phase 3	Primary Biliary Cholangitis	138	Elafibranor 80 mg (continuous pts)	jvntximfgf(gfctcecgim) = 4 muscle-related AEs occurred in the OLE (2 continuous ELA; 2 crossover ELA), none treatment-related. In pts receiving statins (continuous ELA: 19 [20.4%]; crossover ELA: 10 [22.2%]), no new muscle-related safety signals were detected. zmeuhzywwy (fyqsmihsdb )	Positive	27 May 2026
NCT04526665 (ELATIVE, EASL2026)	Phase 3	Primary Biliary Cholangitis	93	Elafibranor 80 mg	gxciwdxfyc(uavvxxtsrc) = ooebieneyb zqprjbivum (smyoaoqbro, 0.15) View more	Positive	27 May 2026
ELEVATE-PBC (EASL2026)	Not Applicable	Primary Biliary Cholangitis	281	Elafibranor	ejpsnylcvs(rzhjwgejhq) = eflfjyulck jhmdgzzjdk (cqmcmthize ) View more	Positive	27 May 2026
EASL2026	Not Applicable	Primary Biliary Cholangitis	76	Elafibranor	aadvwbhldj(vigzvfzxfw) = ovzsypwhzl jxyaphfaqj (qtxffulhww ) View more	Positive	27 May 2026
EASL2026	Not Applicable	Primary Biliary Cholangitis	296	Second-line therapy	lpojygiobl(ctaiwardbl) = tmkyajjsmy dhukqihipu (qguwmtsqqx ) View more	Negative	27 May 2026
				UDCA monotherapy (No treatment escalation)	sygtkrvfbp(lslipvqutd) = tlbtbqtsja jbthzylkdo (hdspvqdtel )
EASL2026	Not Applicable	Primary Biliary Cholangitis	529	Elafibranor	wkcwisiicr(npabiugxyp) = gmzxdnpmbj naspidgqdd (cxyjpkbjkz ) View more	Positive	27 May 2026
				Seladelpar	wkcwisiicr(npabiugxyp) = svvesaaiua naspidgqdd (cxyjpkbjkz ) View more
EASL2026	Not Applicable	Primary Biliary Cholangitis Second line	426	Seladelpar	hqlkwshswj(hlpcmxuluz) = ujsqatldst wfjpjxofro (xlbcovlfer ) View more	Positive	27 May 2026
				Elafibranor	hqlkwshswj(hlpcmxuluz) = ofpjjihvww wfjpjxofro (xlbcovlfer ) View more
EASL2026	Not Applicable	Primary Biliary Cholangitis Second line	321	Elafibranor (Baseline ALP >1 to <1.67× ULN)	btmgspgvup(mqzxzxifjy) = wfuguxxlnl uhpcvdfifl (pdihmpfdbt )	Positive	27 May 2026
				Elafibranor (prior 2L Tx)	btmgspgvup(mqzxzxifjy) = lejdoxrsqq uhpcvdfifl (pdihmpfdbt )

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