In 2024, three ‘first-in-class’ drugs were approved: aprocitentan, an endothelin-1 (ET-1) receptor antagonist (ERA) for the treatment of uncontrolled arterial hypertension, and the fusion protein sotatercept, and the fixed-dose combination of macitentan and tadalafil (M/T-FDC) for the treatment of pulmonary arterial hypertension (PAH). The gene discussed is EDN1; the disease is pulmonary arterial hypertension.