Susan J. Keam
Springer Nature.
New Zealand
Paediatric Drugs
Paediatr Drugs 2025;
DOI: 10.1007/s40272-025-00692-8
Abstract
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.
Category
Medical Therapy. Efficacy or Lack of Efficacy
Medical Therapy. Adverse Effects or Lack of Adverse Effects
Medical Therapy. Pharmacokinetics and Pharmacology
Review Articles Concerning Pulmonary Vascular Disease
Age Focus: Pediatric Pulmonary Vascular Disease
Fresh or Filed Publication: Fresh (PHresh). Less than 1-2 years since publication
Article Access
Free PDF File or Full Text Article Available Through PubMed or DOI: No