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Related Concept Videos

Treatment for Pulmonary Arterial Hypertension: Endothelin Receptor Antagonists01:18

Treatment for Pulmonary Arterial Hypertension: Endothelin Receptor Antagonists

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Endothelins (ETs) are potent vasoactive peptides critical in the human body's various physiological and pathological processes. One of the most promising therapeutic strategies for treating pulmonary arterial hypertension (PAH) involves counteracting the effects of these endothelins using a class of drugs known as endothelin receptor antagonists.
ETs are synthesized through a complex sequence of enzymatic steps, primarily involving an enzyme referred to as endothelin-converting enzyme...
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Receptor tyrosine kinase inhibitors (TKIs) and calcium channel blockers (CCBs) are two critical categories of drugs employed in the treatment of pulmonary artery hypertension (PAH). PAH is a disease that causes high blood pressure in the pulmonary arteries, resulting in chest pain, fatigue, and shortness of breath.
TKIs, such as imatinib (Gleevec), are particularly effective in tackling the growth and mitogenic factors that become upregulated in PAH patients. These factors contribute to the...
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Prostacyclin receptor agonists are a class of therapeutic agents integral to managing pulmonary arterial hypertension (PAH). These drugs operate by mimicking the action of prostaglandin I2, or PGI2, a naturally occurring compound in the body.
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Macitentan: Pediatric First Approval.

Susan J Keam1

  • 1Springer Nature, Private Bag 65901, Mairangi Bay, Auckland 0754, New Zealand. pdd@adis.com.

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|March 15, 2025
PubMed
Summary
This summary is machine-generated.

Macitentan is now approved for treating pediatric pulmonary arterial hypertension (PAH) in Europe and the UK. This marks the first pediatric approval for the endothelin receptor antagonist, offering new long-term treatment options for children aged 2 to 18.

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Area of Science:

  • Pharmacology
  • Pediatric Medicine
  • Cardiology

Background:

  • Macitentan (Opsumit®) is an established endothelin receptor antagonist (ERA) for pulmonary arterial hypertension (PAH).
  • Previous approvals focused on adult patients, necessitating pediatric-specific data for younger populations.

Purpose of the Study:

  • To summarize the development milestones of macitentan leading to its first pediatric approval.
  • To highlight the significance of this approval for treating pediatric PAH.

Main Methods:

  • Review of clinical development data, focusing on Phase 3 studies in pediatric patients.
  • Analysis of regulatory submissions and approvals in the European Union (EU) and the United Kingdom (UK).

Main Results:

  • Macitentan received its first pediatric approval in the EU in September 2024 for patients aged 2 to <18 years (WHO FC II-III).
  • The drug is also approved in the UK for the same pediatric indication.
  • This approval is based on robust Phase 3 clinical trial data.

Conclusions:

  • The pediatric approval of macitentan represents a significant advancement in PAH treatment for children.
  • It provides a new therapeutic option for long-term management of pediatric PAH.
  • Further research may explore long-term outcomes and broader age ranges.