Mavacamten, sold under the brand name Camzyos, is a

medication Medication (also called medicament, medicine, pharmaceutical drug, medicinal product, medicinal drug or simply drug) is a drug used to medical diagnosis, diagnose, cure, treat, or preventive medicine, prevent disease. Drug therapy (pharmaco ...

used to treat obstructive

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

. Mavacamten is a small-molecule allosteric and cardiac

myosin Myosins () are a Protein family, family of motor proteins (though most often protein complexes) best known for their roles in muscle contraction and in a wide range of other motility processes in eukaryotes. They are adenosine triphosphate, ATP- ...

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 respo ...

(FDA) considers it to be a

first-in-class medication A first-in-class medication is a prototype drug 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 medic ...

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 near the beginning of the package insert for certain prescription drugs, so called because the U.S. Food and Drug Administratio ...

regarding heart failure. Mavacamten reduces the left ventricular ejection fraction ( LVEF) and can cause heart failure due to systolic dysfunction.

History

Mavacamten was granted

orphan drug An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by th ...

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 the Committee for Proprietary Medicinal Products (CPMP), is the European Medicines Agency's committee responsible for elaborating the agency's opinions on all issues regar ...

(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 pharmaceutical products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products ...

(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 primary Executive (government), executive arm of the European Union (EU). It operates as a cabinet government, with a number of European Commissioner, members of the Commission (directorial system, informall ...

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) Secondary amines Benzene derivatives Benzyl compounds Drugs developed by Bristol Myers Squibb Orphan drugs Pyrimidines Pyrimidones Cardiac myosin inhibitors