Olorofim for the treatment of invasive fungal diseases in patients with few or no therapeutic options: a single-arm, open-label, phase 2b study.

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Title: Olorofim for the treatment of invasive fungal diseases in patients with few or no therapeutic options: a single-arm, open-label, phase 2b study.
Authors: Maertens JA; Department of Microbiology, Immunology, and Transplantation, KU Leuven, Leuven, Belgium; Department of Hematology, University Hospitals Leuven, Leuven, Belgium. Electronic address: johan.maertens@uzleuven.be., Thompson GR 3rd; Division of Infectious Diseases, Department of Internal Medicine, University of California Davis Medical Center, Sacramento, CA, USA., Spec A; Division of Infectious Disease, Washington University in St Louis School of Medicine, St Louis, MO, USA., Donovan FM; Valley Fever Center for Excellence, Division of Infectious Diseases, Department of Medicine, University of Arizona, Tucson, AZ, USA., Hammond SP; Division of Infectious Diseases and Division of Hematology and Oncology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA; Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA, USA., Bruns AHW; Department of Internal Medicine and Infectious Diseases, University Medical Center, Utrecht, Netherlands., Rahav G; Infectious Diseases Unit, Chaim Sheba Medical Center, Ariel University, Ariel, Israel., Shoham S; Division of Infectious Diseases, Johns Hopkins University School of Medicine, Baltimore, MD, USA., Johnson R; Department of Medicine, David Geffen School of Medicine, University of California, Los Angeles, CA, USA; Division of Infectious Diseases and Valley Fever Institute, Kern Medical, Bakersfield, CA, USA., Rijnders B; Department of Internal Medicine, Section of Infectious Diseases, and Department of Medical Microbiology and Infectious Diseases, Erasmus MC, University Medical Center, Rotterdam, Netherlands., Schaenman J; Division of Infectious Diseases, David Geffen School of Medicine, University of California, Los Angeles, CA, USA; Department of Medicine, David Geffen School of Medicine, University of California, Los Angeles, CA, USA., Hoenigl M; Division of Infectious Diseases, Department of Internal Medicine, Medical University of Graz, Graz, Austria; Biotechmed-Graz, Graz, Austria., Morrissey CO; Department of Infectious Diseases, Alfred Health, Melbourne, VIC, Australia; Department of Infectious Diseases, School of Translational Medicine, Monash University, Melbourne, VIC, Australia., Mehta SR; Division of Infectious Diseases and Global Public Health, University of California San Diego, San Diego, CA, USA; Infectious Diseases Section, San Diego Veterans Affairs Medical Center, San Diego, CA, USA., Heath CH; Department of Microbiology, PathWest Laboratory Medicine, Fiona Stanley Hospital, Murdoch, WA, Australia; Department of Infectious Diseases, Fiona Stanley Hospital, Murdoch, WA, Australia; Department of Medicine, University of Western Australia, Crawley, WA, Australia., Koehler P; European Confederation of Medical Mycology (ECMM) Excellence Center, Department I of Internal Medicine, Faculty of Medicine and University Hospital Cologne, University of Cologne, Cologne, Germany; Division of Clinical Immunology, Department I of Internal Medicine, Faculty of Medicine and University Hospital Cologne, University of Cologne, Cologne, Germany; Cologne Excellence Cluster on Cellular Stress Responses in Aging-Associated Diseases (CECAD), Institute of Translational Research, Faculty of Medicine and University Hospital Cologne, University of Cologne, Cologne, Germany., Paterson DL; ADVANCE-ID, Saw Swee Hock School of Public Health, National University of Singapore, Singapore; Infectious Diseases Translational Research Programme, Yong Loo Lin School of Medicine, National University of Singapore, Singapore., Slavin MA; Department of Infectious Diseases and Sir Peter MacCallum Department of Oncology, Peter MacCallum Cancer Centre, Melbourne, VIC, Australia; Victorian Infectious Diseases Service, Royal Melbourne Hospital and The National Centre for Infections in Cancer, University of Melbourne, Melbourne, VIC, Australia., Fortún J; Infectious Diseases Department, Hospital Ramón y Cajal, Madrid, Spain; CIBER de Enfermedades Infecciosas (CIBERINFEC), Madrid, Spain; Instituto Ramón y Cajal de Investigación Sanitaria (IRYCIS), Madrid, Spain; Facultad de Medicina, Universidad de Alcalá, Madrid, Spain., Nguyen MH; University of Pittsburgh, Pittsburgh, PA, USA., Patterson TF; Department of Medicine, Division of Infectious Diseases, University of Texas Health San Antonio, San Antonio, TX, USA., Uspenskaya O; Leningrad Regional Clinical Hospital, St Petersburg, Russia., Van de Veerdonk FL; Department of Internal Medicine, Radboud University Medical Center, Nijmegen, Netherlands; Radboudumc-CWZ Center of Expertise for Mycology, Nijmegen, Netherlands., Verweij PE; Department of Medical Microbiology, Radboud University Medical Center, Nijmegen, Netherlands; Radboudumc-CWZ Center of Expertise for Mycology, Nijmegen, Netherlands., Aoun M; Infectious Diseases Department, Institut Jules Bordet, Brussels, Belgium., Georgala A; Infectious Diseases Department, Institut Jules Bordet, Brussels, Belgium., Alexander BD; Department of Medicine (Infectious Diseases) and Department of Pathology, Duke University, Durham, NC, USA., Chayakulkeeree M; Division of Infectious Diseases and Tropical Medicine, Department of Medicine, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand., Mehra V; Department of Haematological Medicine, King's College Hospital, London, UK., Miceli MH; Department of Internal Medicine, Division of Infectious Diseases, University of Michigan, Ann Arbor, MI, USA., Sikka MK; Department of Medicine, Division of Infectious Diseases, Oregon Health & Science University, Portland, OR, USA., Solé A; Lung Transplant Unit and Adult Cystic Fibrosis Unit, Hospital Universitari i Politecnic La Fe, Valencia, Spain; Department of Medicine, Division of Pneumology, Universitat de Valencia, Valencia, Spain., Walsh TJ; Center for Innovative Therapeutics and Diagnostics, Richmond, VA, USA; Transplantation-Oncology Infectious Diseases Program, Division of Infectious Diseases, Weill Cornell Medicine, New York, NY, USA., Aguado JM; Unit of Infectious Diseases, Hospital Universitario 12 de Octubre, Instituto de Investigación Sanitaria Hospital 12 de Octubre (imas12), Madrid, Spain; CIBERINFEC, Instituto de Salud Carlos III, Madrid, Spain; Department of Medicine, School of Medicine, Universidad Complutense, Madrid, Spain., Holland SM; Division of Intramural Research, National Institute of Allergy and Infectious Diseases, Bethesda, MD, USA., Moussa M; Hematology and Bone Marrow Transplantation Department, Ain Shams University, Cairo, Egypt., Rautemaa-Richardson R; Mycology Reference Centre Manchester, ECMM Centre of Excellence, Manchester Academic Health Science Centre, Wythenshawe Hospital, Manchester University NHS Foundation Trust, Manchester, UK; Department of Infectious Diseases, Manchester Academic Health Science Centre, Wythenshawe Hospital, Manchester University NHS Foundation Trust, Manchester, UK; Division of Evolution, Infection and Genomics, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, UK., Bazaz R; Department of Infectious Diseases, Manchester Academic Health Science Centre, Wythenshawe Hospital, Manchester University NHS Foundation Trust, Manchester, UK; Division of Evolution, Infection and Genomics, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, UK., Schwartz S; Department of Haematology, Oncology and Cancer Immunology, Campus Benjamin Franklin, Charité-Universitätsmedizin Berlin, corporate member of Freie Universität and Humboldt-Universität zu Berlin, Berlin, Germany., Walsh SR; Division of Infectious Diseases, Brigham & Women's Hospital, Harvard Medical School, Boston, MA, USA., Plate M; Transplantation-Oncology Infectious Diseases Program, Division of Infectious Diseases, Weill Cornell Medicine, New York, NY, USA., Yehudai-Ofir D; Hematology Institute, Rambam Health Care Campus, Haifa, Israel; Ruth and Bruce Rappaport Faculty of Medicine, Technion Israel Institute of Technology, Haifa, Israel., Brüggemann RJ; Department of Pharmacy, Radboud University Medical Center, Nijmegen, Netherlands; Radboudumc-CWZ Center of Expertise for Mycology, Nijmegen, Netherlands., Cornely OA; European Confederation of Medical Mycology (ECMM) Excellence Center, Department I of Internal Medicine, Faculty of Medicine and University Hospital Cologne, University of Cologne, Cologne, Germany; Division of Infectious Diseases, Department I of Internal Medicine, Faculty of Medicine and University Hospital Cologne, University of Cologne, Cologne, Germany; Cologne Excellence Cluster on Cellular Stress Responses in Aging-Associated Diseases (CECAD), Institute of Translational Research, Faculty of Medicine and University Hospital Cologne, University of Cologne, Cologne, Germany; German Centre for Infection Research (DZIF), partner site Bonn-Cologne, Cologne, Germany., Ostrosky-Zeichner L; University of Texas Health Science Center at Houston, Houston, TX, USA., Vazquez JA; Department of Medicine, Division of Infectious Diseases, Medical College of Georgia, Augusta University, Augusta, GA, USA., White PL; Public Health Wales Mycology Regional Reference Laboratory, University Hospital of Wales, Cardiff, UK; Centre for Trials Research, Division of Infection and Immunity, Cardiff University, Cardiff, UK., Cornelissen K; F2G, Nether Alderley, Macclesfield, UK., Ross GG; F2G, Nether Alderley, Macclesfield, UK., Fitton L; F2G, Nether Alderley, Macclesfield, UK., Dane A; DaneStat Consulting, Macclesfield, UK., Zinzi D; F2G, Nether Alderley, Macclesfield, UK., Rex JH; F2G, Nether Alderley, Macclesfield, UK., Chen SC; Centre for Infectious Diseases and Microbiology Laboratory Services, Institute for Clinical Pathology and Medical Research, New South Wales Health Pathology, Westmead Hospital, Sydney, NSW, Australia; Department of Infectious Diseases, Westmead Hospital, Sydney, NSW, Australia; Faculty of Medicine and Health, University of Sydney, Sydney, NSW, Australia.
Source: The Lancet. Infectious diseases [Lancet Infect Dis] 2025 Nov; Vol. 25 (11), pp. 1177-1188. Date of Electronic Publication: 2025 Jun 18.
Publication Type: Journal Article; Clinical Trial, Phase II; Multicenter Study
Journal Info: Publisher: Elsevier Science Country of Publication: United States NLM ID: 101130150 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1474-4457 (Electronic) Linking ISSN: 14733099 NLM ISO Abbreviation: Lancet Infect Dis Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1474-4457
DOI:10.1016/S1473-3099(25)00224-5