PULPulmonary Circulation2045-8940SAGE PublicationsSage UK: London, England10.1177/204589402094149410.1177_2045894020941494Review ArticleRepurposing of medications for pulmonary arterial hypertensionhttps://orcid.org/0000-0002-3969-6143ToshnerMark1SpiekerkoetterEdda2BogaardHarm3HansmannGeorg4NikkhoSylvia5PrinsKurt W.6Department of Medicine, University of Cambridge, Cambridge, UKDepartment of Medicine, Stanford University School of Medicine, Stanford, CA, USADepartment of Pulmonary Medicine, Vrije Universiteit Amsterdam, Amsterdam, NetherlandsDepartment of Pediatric Cardiology and Critical Care, Hannover Medical School, Hannover, GermanyBayer Pharmaceuticals, Clinical Development Pulmonology, Berlin, GermanyLillehei Heart Institute, University of Minnesota, Minneapolis, MN, USAMark Toshner, Department of Medicine, University of Cambridge, Cambridge, UK. Email: mark.toshner@nhs.net Kurt W. Prins, Cardiovascular Division, Lillehei Heart Institute, University of Minnesota, Minneapolis, MN, USA. Email: prin0088@umn.edu18112020104204589402094149417620202062020© The Author(s) 20202020SAGE Publications Ltd, or Pulmonary Vascular Research Institute, unless otherwise noted. Manuscript content on this site is licensed under Creative Commons Licenseshttps://creativecommons.org/licenses/by-nc/4.0/Creative Commons Non Commercial CC BY-NC: This article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 License (https://creativecommons.org/licenses/by-nc/4.0/) which permits non-commercial use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access pages (https://us.sagepub.com/en-us/nam/open-access-at-sage).

This manuscript on drug repurposing incorporates the broad experience of members of the Pulmonary Vascular Research Institute’s Innovative Drug Development Initiative as an open debate platform for academia, the pharmaceutical industry and regulatory experts surrounding the future design of clinical trials in pulmonary hypertension. Drug repurposing, use of a drug in a disease for which it was not originally developed, in pulmonary arterial hypertension has been a remarkable success story, as highlighted by positive large phase 3 clinical trials using epoprostenol, bosentan, iloprost, and sildenafil. Despite the availability of multiple therapies for pulmonary arterial hypertension, mortality rates have modestly changed. Moreover, pulmonary arterial hypertension patients are highly symptomatic and frequently end up on parental therapy and lung transplant waiting lists. Therefore, an unmet need for new treatments exists and drug repurposing may be an important avenue to address this problem.

pulmonary hypertensionpreclinical studiesdrug repurposingcover-dateOctober-December 2020typesetterts2