AI Article Synopsis
- Elafibranor (IQIRVO) is a new medication developed for treating liver diseases such as primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC).
- It received accelerated approval in the US on June 10, 2024, for PBC treatment, particularly for patients who don't respond well to udca or can’t take it.
- The article highlights key milestones in elafibranor's development leading to its first approval for PBC, along with its positive opinion in Europe.
Article Abstract
Elafibranor (IQIRVO) is a first-in-class peroxisome proliferator-activated receptor (PPAR) agonist being developed by Ipsen, under license from Genfit, for the treatment of primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC). On 10 June 2024, elafibranor received accelerated approval based on reduction of alkaline phosphatase (ALP) in the USA for the treatment of PBC in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA. Elafibranor has also received a positive opinion in the EU. This article summarizes the milestones in the development of elafibranor leading to this first approval for PBC.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1007/s40265-024-02075-8 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
PPAR agonists for the treatment of cholestatic liver diseases: Over a decade of clinical progress.
Hepatol Commun
January 2025
Department of Biomedical and Pharmaceutical Sciences, College of Pharmacy, University of Rhode Island, Kingston, Rhode Island, USA.
Article Synopsis
- Primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC) are liver diseases that damage bile ducts, leading to bile accumulation, liver injury, and increased risk of liver failure; current treatments are limited, especially for PSC.
- Ursodeoxycholic acid is the first-line treatment for PBC, but many patients do not respond fully, indicating a need for better therapies; pruritus (itching) is a common and severe symptom due to cholestasis that is often inadequately treated.
- The review examines the use of PPAR agonists, a type of medication that targets liver metabolism, as potential second-line treatments for PBC and PSC, highlighting recent FDA approvals for
Review of Current and Upcoming Second-Line Treatments for Primary Biliary Cholangitis.
Dig Dis Sci
December 2024
Department of Medicine and Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
Background: Treatment for primary biliary cholangitis (PBC) was defined by its singular relationship with ursodeoxycholic acid (UDCA) for decades. However, nearly 40% of patients fail to achieve adequate biochemical response with UDCA, necessitating second-line therapies.
Aims: The aim of our review was to assess the efficacy and safety of second-line therapies for PBC from phase three trials.
Elafibranor: First Approval.
Drugs
September 2024
Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand.
Article Synopsis
- Elafibranor (IQIRVO) is a new medication developed for treating liver diseases such as primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC).
- It received accelerated approval in the US on June 10, 2024, for PBC treatment, particularly for patients who don't respond well to udca or can’t take it.
- The article highlights key milestones in elafibranor's development leading to its first approval for PBC, along with its positive opinion in Europe.
Evaluating the safety and efficacy of seladelpar for adults with primary biliary cholangitis.
Expert Opin Pharmacother
August 2024
Division of Gastroenterology and Hepatology, Henry Ford Health, Detroit, MI, USA.
Article Synopsis
- Seladelpar (MBX-8025) is a targeted PPAR-δ agonist currently being tested in Phase 3 trials for treating primary biliary cholangitis (PBC), with a once-daily dosage.
- The review discusses existing PBC treatments and highlights clinical trial results that show seladelpar's safety, effectiveness, and its positive impact on symptoms, including significant improvements in pruritus and overall quality of life.
- In trials, seladelpar met a key treatment benchmark in 61.7% of patients compared to 20% in the placebo group, indicating promising advancements for PBC therapy.
Efficacy and Safety of Elafibranor in Primary Biliary Cholangitis.
N Engl J Med
February 2024
From Liver Institute Northwest, Seattle (K.V.K.); the Division of Gastroenterology and Hepatology, UC Davis School of Medicine, Sacramento (C.L.B.); Schiff Center for Liver Diseases, University of Miami, Miami (C.L.); the Department of Gastroenterology, Ege University Faculty of Medicine, İzmir, Turkey (U.S.A.); Gastroenterology and Hepatology Division, Hospital de Clinicas de Porto Alegre, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil (M.R.A.-S.); Medicina Interna Metabolica, Baggiovara Hospital, Azienda Ospedaliero-Universitaria di Modena and Università di Modena e Reggio Emilia, Modena (P.A.), and the Division of Gastroenterology, Center for Autoimmune Liver Diseases, Department of Medicine and Surgery, University of Milano-Bicocca, and the European Reference Network on Hepatological Diseases (ERN RARE-LIVER), Fondazione IRCCS San Gerardo dei Tintori, Monza (P.I.) - all in Italy; Departamento de Gastroenterología, Escuela de Medicina, Pontificia Universidad Catolica de Chile, Santiago (M.A.), and Sección de Gastroenterología, Hospital San Juan de la Serena, Coquimbo (J.M. Valera) - both in Chile; the Reference Center for Inflammatory Biliary Disease and Autoimmune Hepatitis, European Reference Network RARE-LIVER, Saint-Antoine Hospital and Research Center, AP-HP, Sorbonne University, Paris (C.C.), GENFIT, Loos (J.-M.G., D.R., B.T.), and Ipsen, Boulogne-Billancourt (S.M.) - all in France; the Department of Gastroenterology and Hepatology, Antwerp University Hospital, and InflaMed Center of Excellence, Laboratory of Experimental Medicine and Paediatrics, Faculty of Medicine and Health Sciences, Antwerp University - both in Antwerp, Belgium (S.M.F.); the Institute of Liver Studies, King's College Hospital NHS Foundation Trust, London (M.A.H.), the Institute of Cellular Medicine and NIHR Newcastle Biomedical Research Center, Newcastle University, Newcastle Upon Tyne (D.J.) - all in the United Kingdom; the Department of Gastroenterology and Hepatology, Mediclinic Durbanville, and Tiervlei Trial Centre - both in Cape Town, South Africa (F.C.K.); the Texas Liver Institute, University of Texas Health, San Antonio (E.L.), the Division of Digestive and Liver Diseases, University of Texas Southwestern Medical Center, Dallas (M.J.M.), and the Departments of Medicine and Surgery, Baylor College of Medicine, Houston (J.M. Vierling) - all in Texas; the Liver Institute of Virginia, Bon Secours Mercy Health, Richmond (M.L.S.); the Liver Unit, Department of Medicine, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada (M.G.S.); Liver Unit, European Reference Network RARE-LIVER, Hospital Universitari Vall d'Hebron, Universitat Autònoma de Barcelona, CiberEhd, Barcelona (V.V.); Hepatic Autoimmunity Unit, Hospital Italiano de Buenos Aires, Buenos Aires (A.V.); GENFIT (C.A., J.D.) and Ipsen (B.M., J.S., C.O.Z.) - both in Cambridge, MA; and the Metabolic Liver Research Program, University Medical Center of the Johannes Gutenberg University Mainz, Mainz, and the Department of Medicine II, Saarland University and Saarland University Medical Center, Homburg - both in Germany (J.M.S.).
Article Synopsis
- Primary biliary cholangitis (PBC) is a chronic liver disease affecting bile ducts, and the effectiveness of elafibranor, a dual PPAR α and δ agonist, in treating PBC was investigated through a clinical trial.
- In a phase 3, double-blind trial with 161 participants, those treated with elafibranor showed a significant biochemical response (51%) compared to only 4% in the placebo group, indicating substantial improvement in liver function.
- While 15% of elafibranor-treated patients normalized their alkaline phosphatase levels, the results for reducing itching (pruritus) between the elafibranor and placebo groups were not statistically
Want AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!