Drug
Mavacamten is a cardiac myosin inhibitor used in symptomatic obstructive hypertrophic cardiomyopathy.
Sources: FDA label · 7 registered trials · 2 guideline recommendations
FDA-approved indications
Source: Camzyos label (DailyMed), label dated Apr 17, 2025
Guideline position
“Results will surely change clinical practice. Your presentation and your study remind us that there's a whole other world of individuals who need their own studies in these broader areas, and that's our pediatric populations. [It's] a rare but compelling contribution that I…”
Expert labels are toward SCOUT-HCM's result, from named public quotes (n=4). As of Apr 5, 2026.
For patients with obstructive HCM who have persistent symptoms attributable to LVOTO despite beta blockers or nondihydropyridine calcium channel blockers, adding a myosin inhibitor (adult patients only), or disopyramide (in combination with an atrioventricular nodal blocking agent), or SRT performed at experienced centers, is recommended.
In patients with HCM who develop persistent systolic dysfunction (LVEF <50%), cardiac myosin inhibitors should be discontinued.
2 recommendations name a dose and are left to the guideline.
FDA actions
Dosing is left to the label.
Primary results of 2 trials behind the evidence on mavacamten, as each trial’s own publication reports them.
mavacamten vs placebo · 112 patients · J Am Coll Cardiol 2022
Primary outcome: the composite of the proportion of patients proceeding with SRT or who remained guideline-eligible after 16 weeks' treatment
“difference (58.9%; 95% CI: 44.0%-73.9%; P < 0.001)”
mavacamten vs placebo · 251 patients · Lancet 2020
Primary outcome: a 1·5 mL/kg per min or greater increase in peak oxygen consumption (pVO2) and at least one NYHA class reduction or a 3·0 mL/kg per min or greater pVO2 increase without NYHA class worsening
“difference +19·4%, 95% CI 8·7 to 30·1; p=0·0005”
Results are quoted from each trial’s primary publication abstract (PubMed); the trial name links to the paper. Ratios below 1 favour the intervention.
Registered trials with mavacamten in an arm, past phase 1 with at least 50 participants: the largest in each indication, newest first. Trials that give it only as the comparator are tagged; trials that give it in every arm are listed last.
NCT07815301 · Not yet recruiting · 286 participants · started 2026
NCT07004972 · Phase 4 · Active not recruiting · 50 participants · started 2025
A Long-Term Safety Extension Study of Mavacamten (MYK-461) in Adults With Hypertrophic Cardiomyopathy Who Have Completed the MAVERICK-HCM (MYK-461-006) or EXPLORER-HCM (MYK-461-005) Trials (MAVA-LTE)
NCT03723655 · Phase 2/3 · Completed · 314 participants · started 2018
Clinical Study to Evaluate Mavacamten (MYK-461) in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy
NCT03470545 · Phase 3 · Completed · 251 participants · started 2018
NCT06112743 · Phase 4 · Active not recruiting · 63 participants · started 2024
A Study of Mavacamten in Non-Obstructive Hypertrophic Cardiomyopathy
NCT05582395 · Phase 3 · Terminated · 580 participants · started 2022
A Study to Evaluate Mavacamten in Adults With Symptomatic Obstructive HCM Who Are Eligible for Septal Reduction Therapy
NCT04349072 · Phase 3 · Completed · 112 participants · started 2020
Source: ClinicalTrials.gov registry records.
Class membership: Synapse curated drug classes
A clinical question appears here only when its condition matches this topic, an outcome and a comparator were extracted from the studies, at least two studies and a patient count are on file, and the certainty is low or higher.
Supports benefit, no benefit shown, and mixed describe the direction of the measured effect. No benefit shown means the evidence does not support a benefit. It is not a finding of harm. Too little evidence means the question did not clear that bar.
Study count, RCT count, and patient count are separate. The patient total adds only studies that are linked into a study family and whose sample size is not flagged as implausible. A reversal is listed only when at least one randomized trial sits on each side of the change.
The overview is drafted from the guideline recommendations, trials, papers, and questions on this page, and every sentence has to cite one of them. It stays unverified until an NPI-verified clinician or a Synapse admin approves it. Approving records that person's name.
Mechanistic diversity within RLC-dependent myosin ATPase inhibitors differentiates EDG-7500, a diastolic-selective cardiac sarcomere modulator
Most-cited researchers publishing on Mavacamten.
