Mavacamten

Mavacamten, sold under the brand name Camzyos, is a medication used to treat obstructive hypertrophic cardiomyopathy.^[7][8]

Mavacamten

Clinical data
Trade names	Camzyos
Other names	MYK-461
AHFS/Drugs.com	Monograph
MedlinePlus	a622047
License data	US DailyMed: Mavacamten
Pregnancy category	AU: D[1]
Routes of administration	By mouth
Drug class	Cardiac myosin inhibitor
ATC code	C01EB24 (WHO)
Legal status
Legal status	AU: S4 (Prescription only)[1][2] BR: Class C1 (Other controlled substances)[3] CA: ℞-only[4][5][6] US: ℞-only[7] EU: Rx-only[8]
Identifiers
IUPAC name 6-[[(1S)-1-phenylethyl]amino]-3-propan-2-yl-1H-pyrimidine-2,4-dione
CAS Number	1642288-47-8
PubChem CID	117761397
DrugBank	DB14921
UNII	QX45B99R3J
KEGG	D12265
Chemical and physical data
Formula	C15H19N3O2
Molar mass	273.336 g·mol−1
3D model (JSmol)	Interactive image
SMILES CC(C)N1C(=O)NC(N[C@@H](C)C2=CC=CC=C2)=CC1=O
InChI InChI=1S/C15H19N3O2/c1-10(2)18-14(19)9-13(17-15(18)20)16-11(3)12-7-5-4-6-8-12/h4-11,16H,1-3H3,(H,17,20)/t11-/m0/s1 Key:RLCLASQCAPXVLM-NSHDSACASA-N

Mavacamten is a small-molecule allosteric ^[9] and cardiac myosin inhibitor.^[7] It was developed by MyoKardia, a subsidiary of Bristol Myers Squibb.^[10] In clinical studies, mavacamten has demonstrated significant efficacy in reducing cardiac muscle contractility by targeting the sarcomere hypercontractility that is one of the characteristics of hypertrophic cardiomyopathy and inhibits excessive myosin actin cross-bridge formation, shifting the overall myosin population towards an energy-sparing, recruitable, super-relaxed state.^[7]

Mavacamten was approved for medical use in the United States in April 2022.^{[7][11][12][13]} The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.^[14][15]

Medical uses

Mavacamten is indicated for the treatment of adults with symptomatic New York Heart Association class II-III obstructive hypertrophic cardiomyopathy to improve functional capacity and symptoms.^[7]

Adverse effects

The US prescribing information for mavacamten contains a boxed warning regarding heart failure. Mavacamten reduces the left ventricular ejection fraction (LVEF) and can cause heart failure due to systolic dysfunction.^[7]

History

Mavacamten was granted orphan drug designation by the US Food and Drug Administration (FDA).^[16]

Society and culture

Legal status

On 26 April 2023, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Camzyos, intended for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM).^[17] The applicant for this medicinal product is Bristol-Myers Squibb Pharma EEIG.^[17] In June 2023, the European Commission approved Mavacamten.^[8][18][19]

Mavacamten is approved for use in the US,^[20] Canada,^[21] Australia,^[22] South Korea,^[23] Singapore,^[24] Switzerland,^[25] Brazil^[26] and Macau.^[27]

Names

Mavacamten is the international nonproprietary name (INN).^[28]