FDA approved Camzyos (mavacamten) for Symptomatic Obstructive Hypertrophic CardiomyopathyFDA approved New Daily Pill for a Type of Heart Condition

Mavacamten is a cardiac myosin inhibitor. The exact mechanism by which it improves functional capacity and symptoms in obstructive HCM is not fully described in the label, but it is thought to reduce excessive cardiac muscle contractility.

Camzyos is indicated for the treatment of adults with symptomatic NYHA class II-III obstructive hypertrophic cardiomyopathy (HCM) to improve functional capacity and symptoms. The safety and effectiveness in pediatric patients have not been established.

The recommended starting dose is 5 mg orally once daily without regard to food. Subsequent doses may be 2.5 mg, 5 mg, 10 mg, or 15 mg once daily, with a maximum recommended dose of 15 mg once daily. Dosage must be individualized based on clinical status and echocardiographic assessment of patient response, including LVEF and Valsalva LVOT gradient. Initiation or up-titration is not recommended in patients with LVEF <55%. If LVEF <50% occurs, interrupt treatment. Missed doses should be taken as soon as possible, but two doses should not be taken on the same day. Capsules should be swallowed whole. Concomitant use with weak to moderate CYP2C19 or moderate to strong CYP3A4 inhibitors requires dose adjustments or interruption as detailed in the label.

The efficacy of Camzyos was evaluated in EXPLORER-HCM (NCT-03470545), a Phase 3, double-blind, randomized, placebo-controlled, multicenter, international, parallel-group trial in 251 adults with symptomatic NYHA class II and III obstructive HCM, LVEF ≥55%, and LVOT peak gradient ≥50 mmHg at rest or with provocation. Patients on dual therapy with beta blocker and calcium channel blocker treatment or monotherapy with disopyramide or ranolazine were excluded. Patients with known infiltrative or storage disorders causing cardiac hypertrophy that mimicked obstructive HCM were also excluded. The primary endpoint and specific results are not detailed in the provided label text. Trial data not available in label.

Camzyos carries a boxed warning for heart failure due to systolic dysfunction. It is contraindicated in pregnancy and in patients with known hypersensitivity to mavacamten or any excipients. Warnings and precautions include the need for regular LVEF and Valsalva LVOT gradient assessment, risk of heart failure, and potential for drug interactions. Confirm absence of pregnancy and usage of effective contraception in females of reproductive potential. Avoid use in patients with LVEF <55% at initiation. Delay dose increases during intercurrent illness or arrhythmia that may impair systolic function.

Camzyos is the first FDA-approved cardiac myosin inhibitor for obstructive HCM. It offers an oral treatment option for adults with symptomatic NYHA class II-III obstructive HCM to improve functional capacity and symptoms. Its use requires careful patient selection and monitoring due to the risk of heart failure and the need for dose titration based on echocardiographic parameters.