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FDA approves new drug to improve heart function in adults with rare heart condition

Apr 29 2022


FDA has approved Camzyos (mavacamten) capsules to treat adults with symptomatic New York Heart Association (NYHA)External Link Disclaimer class II-III obstructive hypertrophic cardiomyopathy (oHCM) to improve exercise capacity and symptoms.



Disease or Condition


oHCM is a rare disease that occurs when the heart muscle thickens, mostly in the septum (the heart wall that separates its right and left sides) and obstructs blood flow from the heart to the rest of the body.


The severity of oHCM varies, but it can cause serious symptoms and death. Symptoms include palpitations (the sensation the heart is pounding), shortness of breath, swelling of the legs, and decreased exercise capacity, which significantly impact daily activities and quality of life.



Effectiveness


The effectiveness and safety of Camzyos to treat NYHA class II-III oHCM was studied in adults who were randomly assigned to receive Camzyos or a placebo for 30 weeks. At the end of the study, 37% of participants treated with Camzyos improved on an endpoint measuring exercise capacity and symptoms, compared to 17% of participants in the placebo group.



Safety


Camzyos reduces contraction of the heart muscle, which can cause heart failure or totally block functioning of the ventricles (lower chambers of the heart). Patients who have a serious intercurrent illness (such as a serious infection) or arrhythmia (atrial fibrillation or other uncontrolled fast heart rhythm abnormality) are at greater risk of developing impaired heart muscle contraction and heart failure with Camzyos.


Because of the risk for heart failure, patients treated with Camzyos must be monitored with echocardiograms, an imaging test that shows how well the heart is working. Patients must also avoid certain prescription and over-the-counter medicines that interfere with the metabolism (breakdown) of Camzyos.


Because of the potential for developing symptomatic or life-threatening heart failure if monitoring is not followed or if Camzyos is used with medicines that impair its metabolism, Camzyos is only available through a restricted program called the Camzyos Risk Evaluation and Mitigation Strategy (REMS). The REMS program helps ensure safe use by lowering the risk of heart failure due to impaired heart muscle contraction.


Camzyos received breakthrough therapy designation and orphan drug designation for this indication.




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