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Phase 3 Open-Label Study Evaluating the Efficacy and Safety of Mavacamten in Japanese Adults With Obstructive

Hiroaki Kitaoka1, Masaki Ieda2, Mio Ebato3

  • 1Department of Cardiology and Geriatrics, Kochi Medical School, Kochi University.

Circulation Journal : Official Journal of the Japanese Circulation Society
|November 6, 2024
PubMed

Insights

Mavacamten improved symptoms and cardiac function in Japanese patients with obstructive hypertrophic cardiomyopathy (HCM). The cardiac myosin inhibitor demonstrated a favorable safety profile, similar to previous trials.

Area of Science:

  • Cardiology
  • Pharmacology

Background:

  • Mavacamten, a cardiac myosin inhibitor, showed efficacy in symptomatic obstructive hypertrophic cardiomyopathy (HCM) in the EXPLORER-HCM trial.
  • The efficacy and safety of mavacamten in Japanese patients with obstructive HCM were previously unclear.

Purpose of the Study:

  • To evaluate the efficacy and safety of mavacamten in Japanese patients with symptomatic obstructive HCM.
  • To assess mavacamten's impact on left ventricular outflow tract (LVOT) gradient, cardiac biomarkers, and clinical symptoms.

Main Methods:

  • Phase 3, single-arm study (HORIZON-HCM) in Japanese patients with symptomatic obstructive HCM.
  • Mavacamten initiated at 2.5 mg, with individualized dose titration based on LVOT gradient and LVEF over 30 weeks.
  • 38 patients treated, 36 completed the primary analysis period.

Main Results:

  • Significant improvement in postexercise LVOT gradient (mean change -60.7 mmHg) after 30 weeks.
  • Observed improvements in N-terminal pro B-type natriuretic peptide, NYHA class, and KCQ-23 CSS.
  • Mean LVEF remained ≥74% throughout the study; treatment was well tolerated.

Conclusions:

  • Mavacamten demonstrated efficacy in Japanese patients with obstructive HCM, comparable to EXPLORER-HCM findings.
  • Improvements in LVOT gradients, cardiac biomarkers, and symptoms were noted.
  • Mavacamten was well tolerated, with no new safety concerns identified.
Abstract

Keywords:
EfficacyHypertrophic cardiomyopathyMavacamtenSafety

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