Mavacamten
drugOn this page
Also known as CamzyosMyk-461SAR-439152Marvacamten
Summary
Mavacamten (CHEMBL4297517) is an approved small molecule (ATC C01EB24); indicated across 3 conditions including hypertrophic cardiomyopathy and cardiovascular disorder.
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Small molecule
- ATC class: C01EB24
- Indications: 3 conditions
- Clinical trials: 39
- Chemistry: 273.33 Da · C15H19N3O2
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL4297517 |
| Name | Mavacamten |
| Type | Small molecule |
| Max phase | 4 |
| FDA approved | yes |
| PubChem CID | 117761397 |
| ATC | C01EB24 |
| Molecular formula | C15H19N3O2 |
| Molecular weight | 273.33 |
| InChIKey | RLCLASQCAPXVLM-NSHDSACASA-N |
SMILES: C[C@@H](C1=CC=CC=C1)NC2=CC(=O)N(C(=O)N2)C(C)C
IUPAC name: 6-[[(1S)-1-phenylethyl]amino]-3-propan-2-yl-1H-pyrimidine-2,4-dione
Also known as: Camzyos, Mavacamten, Myk-461, MYK-461, SAR-439152, MAVACAMTEN, Marvacamten
Patent coverage: 84 distinct patent families (208 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.
Targets
Targets
Broader ChEMBL bioactivity targets: 2 (assay-derived). Sample: Cytochrome P450 3A4, Cytochrome P450 2B6.
Bioactivity
ChEMBL activities: 2 potent at pChembl ≥ 5 of 2 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):
| Target | pChembl | Type | Value | Unit | Activity ID |
|---|---|---|---|---|---|
| CYP3A4 | 5.66 | EC50 | 2200 | nM | CHEMBL_ACT_23263417 |
| CYP2B6 | 5.29 | EC50 | 5100 | nM | CHEMBL_ACT_23263418 |
Target pathways
No target-pathway data for this drug (no mapped target genes).
Indications & clinical
Indications
3 indications (2 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).
| Indication | Trial phase | MONDO | EFO |
|---|---|---|---|
| hypertrophic cardiomyopathy | 4 | MONDO:0005045 | EFO:0000538 |
| cardiovascular disorder | 4 | MONDO:0004995 | EFO:0000319 |
| heart failure | 2 | MONDO:0005252 | EFO:0003144 |
Clinical trials
Total trials: 39.
Phase distribution
| Phase | Trials |
|---|---|
| Not specified | 18 |
| PHASE1 | 7 |
| PHASE3 | 6 |
| PHASE2 | 4 |
| PHASE4 | 3 |
| PHASE2/PHASE3 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT06112743 | PHASE4 | ACTIVE_NOT_RECRUITING | A Study to Evaluate Mavacamten Impact on Myocardial Structure in Participants With Symptomatic Obstructive Hypertrophic Cardiomyopathy |
| NCT07004972 | PHASE4 | ACTIVE_NOT_RECRUITING | A Study of Mavacamten in Adults With Obstructive Hypertrophic Cardiomyopathy in India (ROVER) |
| NCT07103655 | PHASE4 | NOT_YET_RECRUITING | The Therapeutic Value of Mavacamten in Hypertrophic Cardiomyopathy With Mid-to-Apical Left Ventricular Obstruction |
| NCT06253221 | PHASE3 | ACTIVE_NOT_RECRUITING | A Study to Evaluate Mavacamten in Adolescents With Symptomatic Obstructive Hypertrophic Cardiomyopathy |
| NCT03470545 | PHASE3 | COMPLETED | Clinical Study to Evaluate Mavacamten (MYK-461) in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy |
| NCT03723655 | PHASE2/PHASE3 | COMPLETED | A Long-Term Safety Extension Study of Mavacamten in Adults Who Have Completed MAVERICK-HCM or EXPLORER-HCM |
| NCT04349072 | PHASE3 | COMPLETED | A Study to Evaluate Mavacamten in Adults With Symptomatic Obstructive HCM Who Are Eligible for Septal Reduction Therapy |
| NCT05174416 | PHASE3 | COMPLETED | A Study to Evaluate the Efficacy and Safety of Mavacamten in Chinese Adults With Symptomatic Obstructive HCM |
| NCT05414175 | PHASE3 | COMPLETED | A Study of Mavacamten in Obstructive Hypertrophic Cardiomyopathy |
| NCT05582395 | PHASE3 | COMPLETED | A Study of Mavacamten in Non-Obstructive Hypertrophic Cardiomyopathy |
| NCT02842242 | PHASE2 | COMPLETED | A Phase 2 Open-label Pilot Study Evaluating MYK-461 in Subjects With Symptomatic Hypertrophic Cardiomyopathy and Left Ventricular Outflow Tract Obstruction |
| NCT03442764 | PHASE2 | COMPLETED | A Phase 2 Study of Mavacamten in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM) |
| NCT03496168 | PHASE2 | COMPLETED | Extension Study of Mavacamten (MYK-461) in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy Previously Enrolled in PIONEER |
| NCT04766892 | PHASE2 | COMPLETED | A Study of Mavacamten in Participants With HFpEF and Elevation of NT-proBNP With or Without Elevation of cTnT |
| NCT02329184 | PHASE1 | COMPLETED | Study Evaluating the Safety, Tolerability and Preliminary Pharmacokinetics and Pharmacodynamics of MYK-461 |
| NCT02356289 | PHASE1 | COMPLETED | Single Ascending Dose Study of MYK-461 in Healthy Volunteers |
| NCT02480296 | PHASE1 | COMPLETED | Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Oral Doses of MYK-461 in Healthy Volunteers |
| NCT05135871 | PHASE1 | COMPLETED | Study Evaluating the Pharmacokinetics of Mavacamten in Healthy Adult Chinese Subjects |
| NCT05320094 | PHASE1 | COMPLETED | A Study to Evaluate the Effect of Activated Charcoal With Sorbitol on the Single-dose of Mavacamten in Healthy Participants |
| NCT05362045 | PHASE1 | COMPLETED | A Study to Evaluate the Effects on the Single-Dose Drug Levels of Mavacamten in Healthy Participants |
| NCT05719805 | PHASE1 | COMPLETED | A Study to Evaluate the Effects of Mavacamten in Healthy Participants |
| NCT05489705 | Not specified | RECRUITING | A Prospective Registry Study to Assess Real-world Patient Characteristics, Treatment Patterns, and Longitudinal Outcomes in Patients Receiving Mavacamten and Other Treatments for Symptomatic Obstructive Hypertrophic Cardiomyopathy (Obstructive-HCM) |
| NCT05939700 | Not specified | RECRUITING | Mavacamten Pregnancy Surveillance Program |
| NCT06023186 | Not specified | RECRUITING | Effect of Mavacamten Treatment on Coronary Flow Reserve in oHCM |
| NCT06146660 | Not specified | RECRUITING | A Study to Assess the Safety of Mavacamten in Korean Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy |
| NCT06372457 | Not specified | ACTIVE_NOT_RECRUITING | COLLIGO-HCM: A Multinational Observational Study of the Real-World Effectiveness of Mavacamten Among Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM) |
| NCT06549608 | Not specified | ACTIVE_NOT_RECRUITING | A Retrospective Cohort Study of Mavacamten Patient Support Program in Canada |
| NCT06551129 | Not specified | RECRUITING | Real-world Patient Reported Outcomes Among Patients Treated With Camzyos |
| NCT06856265 | Not specified | NOT_YET_RECRUITING | Efficacy of Mavacamten Combined With Radiofrequency Ablation in Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy |
| NCT06947590 | Not specified | ACTIVE_NOT_RECRUITING | Efficacy of Mavacamten in Patients With Symptomatic Latent Obstructive Hypertrophic Cardiomyopathy |
| NCT07107373 | Not specified | ACTIVE_NOT_RECRUITING | A Study to Assess the Treatment of Obstructive Hypertrophic Cardiomyopathy (oHCM) With Mavacamten in the US |
| NCT07120776 | Not specified | RECRUITING | Positron Emission Tomography to Assess the Effect of Camzyos on Ischaemia in HOCM: PEACH Trial |
| NCT07361289 | Not specified | RECRUITING | A Study to Assess the Real-World Effectiveness of Mavacamten in Adult Patients With Obstructive Hypertrophic Cardiomyopathy in China |
| NCT07383025 | Not specified | RECRUITING | Mavacamten Post-marketing Surveillance in Patients With Obstructive Hypertrophic Cardiomyopathy in Japan |
| NCT07541833 | Not specified | RECRUITING | Effectiveness and Treatment Patterns of Mavacamten in Patients With Obstructive Hypertrophic Cardiomyopathy in Japan (MANAGE-HCM) |
| NCT07557498 | Not specified | RECRUITING | Mavacamten Outcomes in Hypertrophic Cardiomyopathy (HCM) and the Associated Patient and Physician Experiences in the US |
| NCT06338202 | Not specified | COMPLETED | Real-World Effectiveness of Mavacamten in Canada |
| NCT07168655 | Not specified | COMPLETED | Mavacamten in Obstructive Hypertrophic Cardiomyopathy |
| NCT07529938 | Not specified | COMPLETED | Mavacamten in Adult Patients With Obstructive Hypertrophic Cardiomyopathy |
Clinical evidence (CIViC)
No CIViC predictive evidence (expected for non-precision-medicine drugs).
Pharmacology
Pharmacogenomics
PharmGKB dosing guidelines (2) — CPIC / DPWG genotype-guided dosing for this drug (drug × pharmacogene):
| Guideline | Source | Gene(s) | Dosing | Recommendation |
|---|---|---|---|---|
| Annotation of RNPGx Guideline for mavacamten and CYP2C19 | RNPGx | CYP2C19 | yes | yes |
| Annotation of DPWG Guideline for mavacamten and CYP2C19 | DPWG | CYP2C19 | yes | yes |
PharmGKB also curates 0 clinical and 1 variant annotation(s) for this drug (gene-keyed; see PharmGKB).
Related molecules
Related molecules
No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).