Safety and efficacy of elafibranor in primary sclerosing cholangitis: The ELMWOOD phase II randomized-controlled trial
For people with primary sclerosing cholangitis (PSC), there is a need for a well-tolerated and effective treatment that will enhance quality of life, prevent disease progression, and improve long-term outcomes. Here, we present results from the double-blind period of the phase II ELMWOOD trial in PSC, wherein elafibranor, a peroxisome proliferator-activated receptor-α/δ agonist, demonstrated a favorable safety profile, provided greater biochemical improvements over 12 weeks compared with placebo, and appeared to stabilize markers of fibrosis and improve pruritus. These findings support larger and longer term investigations of elafibranor to explore its therapeutic potential as a treatment for people with PSC.
Authors
- Antonio Olveira (ORCID: https://orcid.org/0000-0002-2991-4688)
- Kathrin Sprinzl (ORCID: https://orcid.org/0000-0002-0599-3801)
- Douglas Thorburn (ORCID: https://orcid.org/0000-0002-5610-5448)
- Richard Gilroy
- Rachael Swann (ORCID: https://orcid.org/0000-0002-3945-2253)
- Faruq Pradhan (ORCID: https://orcid.org/0000-0002-5249-188X)
- Aldo J. Montaño‐Loza (ORCID: https://orcid.org/0000-0002-2511-7980)
- Mitchell L. Shiffman (ORCID: https://orcid.org/0000-0002-3575-6516)
- George Abouda
- Alessandra Mangia (ORCID: https://orcid.org/0000-0002-2600-3555)
- Velimir A. Luketic (ORCID: https://orcid.org/0000-0002-0256-1456)
- Juan Turnés (ORCID: https://orcid.org/0000-0001-8426-6145)
- Ashis Mukhopadhya
- Helena Cortez‐Pinto (ORCID: https://orcid.org/0000-0002-8537-8744)
- Benjamin F. Miller (ORCID: https://orcid.org/0000-0003-3283-0685)
- Bertus Eksteen (ORCID: https://orcid.org/0000-0001-7165-9214)
- Christopher L. Bowlus (ORCID: https://orcid.org/0000-0002-3906-6811)
- Michael T. Dill (ORCID: https://orcid.org/0000-0002-1926-4204)
- Hugo Gomes da Silva
- Kuldeep Cheent
- Jonathan M. Fenkel (ORCID: https://orcid.org/0000-0003-4612-8416)
- Aude Tavenard
- Antonello Pietrangelo (ORCID: https://orcid.org/0000-0002-7411-935X)
- Isabel Campos‐Varela (ORCID: https://orcid.org/0000-0001-6597-3151)
- Claudia O. Zein
- Natasha Chandok
- Hin Hin Ko (ORCID: https://orcid.org/0000-0002-6433-5956)
- Yiannis Kallis (ORCID: https://orcid.org/0000-0003-4921-8134)
- Paul J. Thuluvath (ORCID: https://orcid.org/0000-0002-4374-4507)
- Cynthia Levy (ORCID: https://orcid.org/0000-0001-5498-6037)
- Münevver Demir (ORCID: https://orcid.org/0000-0002-7050-797X)
- Palak Trivedi (ORCID: https://orcid.org/0000-0002-4009-8087)
- Seema Jaitly
- Alan Bonder (ORCID: https://orcid.org/0000-0001-6821-0416)
- Ira M. Jacobson (ORCID: https://orcid.org/0000-0003-1803-2214)
- Nora Cazzagon (ORCID: https://orcid.org/0000-0002-6937-8664)
- Bhaktasharan Patel
- C. Milligan
- Magdalena Salcedo (ORCID: https://orcid.org/0000-0002-1239-5746)
- Kris V. Kowdley (ORCID: https://orcid.org/0000-0002-8553-3652)
- Marcelo Kugelmas
- Bahri M. Bilir
Institutions
- Intermountain Healthcare (US)
- Goethe University Frankfurt (DE)
- University of Modena and Reggio Emilia (IT)
- Hebron University (PS)
- University of Maryland, Baltimore (US)
- Rocky Mountain Institute (US)
- Beth Israel Deaconess Medical Center (US)
- University of Padua (IT)
- University of Lisbon (PT)
- University of British Columbia (CA)
- University of Miami (US)
- University of Alberta (CA)
- Queen Mary University of London (GB)
- Ipsen (France) (FR)
- Heidelberg University (DE)
- Thomas Jefferson University Hospital (US)
- Hull University Teaching Hospitals NHS Trust (GB)
- Casa Sollievo della Sofferenza (IT)
- Hospital Universitario La Paz (ES)
- Aberdeen Royal Infirmary (GB)
- The Royal Free Hospital (GB)
- University Hospital Heidelberg (DE)
- Frimley Health NHS Foundation Trust (GB)
- Intermountain Medical Center (US)
- Hospital General Universitario Gregorio Marañón (ES)
- NYU Langone Health (US)
- Ipsen (United Kingdom) (GB)
- NIHR Birmingham Liver Biomedical Research Unit (GB)
- Liver Institute Northwest (US)
- Vall d'Hebron Hospital Universitari (ES)
- William Osler Health System (CA)
- Bon Secours Mercy Health (US)
- Bon Secours Liver Institute of Richmond (US)
- University Hospital Frankfurt (DE)
- Rocky Mountain Orthopedics (US)
- Ipsen (United States) (US)
- Istituti di Ricovero e Cura a Carattere Scientifico (IT)
- Queen Elizabeth University Hospital (GB)
- Complejo Hospitalario de Pontevedra (ES)
- NIHR Birmingham Biomedical Research Centre (GB)
- University College London (GB)
- University of Nebraska Medical Center (US)
- Charité - Universitätsmedizin Berlin (DE)
- University of California, Davis (US)
- University of Colorado Colorado Springs (US)
Publication Details
- Journal
- Journal of Hepatology
- Published
- 2025-05-10
- DOI
- https://doi.org/10.1016/j.jhep.2025.04.025
- Citations
- 10
- Primary Topic
- Liver Diseases and Immunity
- Type
- article
- Field-Weighted Citation Impact
- 13.22
Funders
- AstraZeneca
- GlaxoSmithKline
- Sanofi
- Gilead Sciences
- Ipsen Biopharmaceuticals
- NGM Biopharmaceuticals
- Eisai
- Novo Nordisk
- Astellas Pharma
- Ipsen
- Dr. Falk Pharma