Safety, tolerability, pharmacokinetics, and pharmacodynamics of macitentan, an endothelin receptor antagonist, in an ascending multiple-dose study in healthy subjects

J Clin Pharmacol. 2013 Nov;53(11):1131-8. doi: 10.1002/jcph.152. Epub 2013 Sep 4.

Abstract

This multiple-ascending-dose study investigated safety, tolerability, pharmacokinetics, and pharmacodynamics, of macitentan, a new endothelin receptor antagonist (ERA) with sustained receptor binding and enhanced tissue penetration properties compared to other ERAs. Healthy male subjects (n = 32) received once daily oral doses of macitentan (1 - 30 mg) or placebo for 10 days. Administration of macitentan was safe and well tolerated. Macitentan had no effect on bile salts, suggesting an improved liver safety profile. The multiple-dose pharmacokinetics of macitentan were dose-proportional and were characterized by a median tmax and apparent elimination half-life varying from 6.0 to 8.5 and 14.3 to 18.5 hours, respectively, for the different doses and minimal accumulation. ACT-132577, a metabolite with lower potency than macitentan, had a half-life of about 48 hours and accumulated approximately 8.5-fold. Compared to placebo, administration of macitentan caused a dose-dependent increase in plasma ET-1 with maximum effects attained at 10 mg. A small dose-dependent increase in the 6β-hydroxycortisol/cortisol urinary excretion ratio was observed, although there were no statistically significant differences between treatments including placebo. Effects of macitentan on cytochrome P450 enzyme 3A4 should be further evaluated in dedicated studies. The present results support investigation of macitentan in the management of pulmonary arterial hypertension and ET-1-dependent pathologies.

Keywords: endothelin receptor antagonist; healthy subjects; macitentan; pharmacodynamics; pharmacokinetics.

Publication types

  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Bile Acids and Salts / blood
  • Dose-Response Relationship, Drug
  • Double-Blind Method
  • Endothelin A Receptor Antagonists*
  • Endothelin B Receptor Antagonists*
  • Endothelin-1 / blood
  • Familial Primary Pulmonary Hypertension
  • Humans
  • Hydrocortisone / analogs & derivatives
  • Hydrocortisone / blood
  • Hypertension, Pulmonary
  • Male
  • Middle Aged
  • Pyrimidines / administration & dosage*
  • Pyrimidines / adverse effects
  • Pyrimidines / pharmacokinetics
  • Sulfonamides / administration & dosage*
  • Sulfonamides / adverse effects
  • Sulfonamides / pharmacokinetics
  • Young Adult

Substances

  • Bile Acids and Salts
  • Endothelin A Receptor Antagonists
  • Endothelin B Receptor Antagonists
  • Endothelin-1
  • Pyrimidines
  • Sulfonamides
  • 6 beta-hydroxycortisol
  • Hydrocortisone
  • macitentan