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.
Bibliographical noteFunding Information:
We thank Paul Corris for the general supervision of the Innovative Drug Development Initiative and their dedicated support of the Drug Repurposing workstream, as well as Georgie Sutton, PVRI Admin Manager, for her support in proofreading this manuscript.
© The Author(s) 2020.
- drug repurposing
- preclinical studies
- pulmonary hypertension