A Belgian consensus on sotatercept for the treatment of pulmonary arterial hypertension.

Abstract

Pulmonary arterial hypertension (PAH) is a rare disease affecting the small pulmonary vessels, ultimately leading to right ventricular failure and death. Current treatment options target three different pathways (endothelin, nitric oxide/cGMP and prostacyclin pathways). Despite their demonstrated efficacy, these therapies (commonly used in combination) do not cure the disease which is why novel pathways beyond the traditional 'big three' are being developed. Sotatercept is a ligand trap for multiple proteins within the TGF-β superfamily that was recently approved in the US for the treatment of PAH. Unlike currently available therapies, sotatercept has the potential to act as an anti-remodelling agent rather than a vasodilator. The safety and efficacy of subcutaneous (SC) sotatercept have been established in two multicentre, placebo-controlled randomised-controlled trials. The compound has been shown to consistently improve a variety of measurable endpoints, including exercise capacity, haemodynamics, quality of life and delay of clinical worsening. The drug appears to have an acceptable safety profile, although it is associated with an increased risk in developing telangiectasia and biological changes affecting platelet counts and haemoglobin. This study reviews the current evidence on SC sotatercept and provides a Belgian perspective on its place in the future treatment strategy for PAH.