Mavacamten, a Novel Therapeutic Strategy for Obstructive Hypertrophic Cardiomyopathy.
Mattia ZampieriAlessia ArgiròAlberto MarchiMartina BerteottiMattia TargettiAlessandra FornaroAlessia TomberliPierluigi StefànoNiccolò MarchionniIacopo OlivottoPublished in: Current cardiology reports (2021)
Mavacamten is a novel, first-in-class, allosteric inhibitor of cardiac myosin ATPase, which reduces actin-myosin cross-bridge formation, thereby reducing myocardial contractility and improving myocardial energetic consumption in experimental HCM models. Following a successful Phase 2 study, the recently published phase III, placebo-controlled, randomized EXPLORER-HCM trial demonstrated the efficacy and safety of mavacamten in reducing left ventricular outflow tract obstruction and ameliorating exercise capacity, New York Heart Association functional class and health status in patients with obstructive HCM. Mavacamten represents the first agent specifically developed for HCM successfully tested in a Phase III trial, to be registered soon for clinical use, representing a radical change of paradigm in the pharmacological treatment of HCM.
Keyphrases
- hypertrophic cardiomyopathy
- phase iii
- left ventricular
- placebo controlled
- double blind
- open label
- phase ii
- heart failure
- clinical trial
- acute myocardial infarction
- cardiac resynchronization therapy
- study protocol
- mitral valve
- left atrial
- aortic stenosis
- phase ii study
- binding protein
- physical activity
- atrial fibrillation
- combination therapy
- ejection fraction
- cell migration
- locally advanced