Key result
Drug repurposing represents an important avenue to address the unmet need for new treatments in pulmonary arterial hypertension.
Why the study?
Despite available therapies, pulmonary arterial hypertension mortality rates have only modestly changed and patients remain highly symptomatic, leaving an unmet need for new treatments.
Drug repurposing remains an important avenue to address the unmet need for new treatments in pulmonary arterial hypertension.
Supports drug repurposing strategies in pulmonary arterial hypertension; hypothesis-generating and requires validation before practice change.
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.
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Toshner et al. (2020) conducted a review in Pulmonary arterial hypertension. Drug repurposing was evaluated. Drug repurposing represents an important avenue to address the unmet need for new treatments in pulmonary arterial hypertension.
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