Sotatercept in pulmonary arterial hypertension: revolution, risk and the road ahead

Sotatercept, the first approved activin signalling inhibitor (ASI), represents a major advancement in the treatment of pulmonary arterial hypertension (PAH). Met with substantial enthusiasm from patients, caregivers and physicians, sotatercept has the potential to refine, and perhaps even revolutionise, the treatment of PAH. Its novel mechanism of action targets fundamental disease processes, offering the potential of partial disease reversal. Data from a phase 2 study and three phase 3 studies demonstrate robust efficacy. However, unique safety signals have also emerged, both in the clinical trial programme and in post-marketing surveillance. This article summarises the present evidence and outlines steps to optimise the benefit/risk ratio of sotatercept and future ASIs.