A phase 3 trial of a first-in-class, non–FDA-approved agent is encouraging, but questions remain.
Pulmonary arterial hypertension (PAH), while relatively rare, disproportionately affects younger individuals, particularly women, and has a dire prognosis. Although several classes of beneficial therapies are available — including phosphodiesterase-5 inhibitors, soluble guanylate cyclase stimulators, and endothelin-receptor antagonists — morbidity and mortality remain high. This phase 3, manufacturer-sponsored trial (NCT04576988) assessed the efficacy of sotatercept, a compound that acts as a “ligand trap” for selected TGF-β superfamily members, which have been implicated in the pathogenesis of PAH.
Investigators randomized 323 individuals with PAH (median age, 48 years; 79% women; 89% white; 59% with idiopathic PAH; 61% on three agents to t…
Reviewing Author
DisclosuresConsultant/Advisory BoardBristol Myers Squibb; CPC Clinical Research
Grant/Research SupportNational Heart, Lung, and Blood Institute
Editorial BoardsUpToDate; American College of Cardiology Self-Assessment Program (SAP)
Leadership Positions in Professional SocietiesAmerican College of Cardiology (Chair, Innovations Committee)
DisclosuresConsultant/Advisory BoardBristol Myers Squibb; CPC Clinical Research
Grant/Research SupportNational Heart, Lung, and Blood Institute
Editorial BoardsUpToDate; American College of Cardiology Self-Assessment Program (SAP)
Leadership Positions in Professional SocietiesAmerican College of Cardiology (Chair, Innovations Committee)