Macitentan: Pediatric First Approval

Macitentan (Opsumit^®), an endothelin receptor antagonist (ERA) developed by Johnson & Johnson, is well established worldwide as monotherapy or combination therapy for the long-term treatment of pulmonary arterial hypertension (PAH). In September 2024, based on phase 3 clinical data in patients aged < 18 years, macitentan received its first pediatric approval in the EU as monotherapy or in combination for the long-term treatment of PAH in pediatric patients aged 2 years to < 18 years with WHO functional class (FC) II to III. Macitentan has also been approved in the UK for this indication. This article summarizes the milestones in the development of macitentan leading to the first pediatric approval for the long-term treatment of PAH.