Mavacamten, sold under the brand name Camzyos, is a

medication A medication (also called medicament, medicine, pharmaceutical drug, medicinal drug or simply drug) is a drug used to diagnose, cure, treat, or prevent disease. Drug therapy (pharmacotherapy) is an important part of the medical field and re ...

used to treat obstructive

hypertrophic cardiomyopathy Hypertrophic cardiomyopathy (HCM, or HOCM when obstructive) is a condition in which the heart becomes thickened without an obvious cause. The parts of the heart most commonly affected are the interventricular septum and the ventricles. This r ...

. Mavacamten is a small-molecule allosteric and cardiac

myosin Myosins () are a superfamily of motor proteins best known for their roles in muscle contraction and in a wide range of other motility processes in eukaryotes. They are ATP-dependent and responsible for actin-based motility. The first myosin ...

inhibitor. It was developed by MyoKardia, a subsidiary of Bristol Myers Squibb. 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. Mavacamten was approved for medical use in the United States in April 2022. The US

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respon ...

(FDA) considers it to be a

first-in-class medication A first-in-class medication is a pharmaceutical that uses a "new and unique mechanism of action" to treat a particular medical condition. While the Food and Drug Administration's Center for Drug Evaluation and Research tracks first-in-class medicat ...

Medical uses

Mavacamten is

indicated In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis ...

for the treatment of adults with symptomatic New York Heart Association class II-III obstructive hypertrophic cardiomyopathy to improve functional capacity and symptoms.

Adverse effects

The US prescribing information for mavacamten contains a

boxed warning In the United States, a boxed warning (sometimes "black box warning", colloquially) is a type of warning that appears on the package insert for certain prescription drugs, so called because the U.S. Food and Drug Administration specifies that it ...

regarding heart failure. Mavacamten reduces LVEF and can cause heart failure due to systolic dysfunction.

History

Mavacamten was granted

orphan drug An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases. The assignment of ...

designation by the US

(FDA).

Society and culture

Legal status

On 26 April 2023, the

Committee for Medicinal Products for Human Use The Committee for Medicinal Products for Human Use (CHMP), formerly known as Committee for Proprietary Medicinal Products (CPMP), is the European Medicines Agency's committee responsible for elaborating the agency's opinions on all issues regarding ...

(CHMP) of the

European Medicines Agency The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of medicinal products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products or Euro ...

(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). The applicant for this medicinal product is Bristol-Myers Squibb Pharma EEIG. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged. In June 2023, the

European Commission The European Commission (EC) is the executive of the European Union (EU). It operates as a cabinet government, with 27 members of the Commission (informally known as "Commissioners") headed by a President. It includes an administrative body o ...

approved Mavacamten. Mavacamten is approved for use in the US, Canada, Australia, South Korea, Singapore, Switzerland, Brazil and Macau.

Names

Mavacamten is the international nonproprietary name (INN).

References

External links

* {{ClinicalTrialsGov, NCT03470545, Clinical Study to Evaluate Mavacamten (MYK-461) in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy (EXPLORER-HCM) Amines Benzene derivatives Benzyl compounds Drugs developed by Bristol Myers Squibb Orphan drugs Pyrimidines Pyrimidones Cardiac myosin inhibitors