Emerging targeted therapies, including the newly FDA-approved cardiac myosin inhibitor mavacamten, provide novel disease-modifying treatment options for patients with hypertrophic cardiomyopathy.
The review highlights the transition in HCM management from purely symptom-directed therapies to emerging targeted, potentially disease-modifying treatments.
Hypertrophic cardiomyopathy (HCM) is a genetic disorder caused by pathogenic variants in sarcomeric genes, leading to left ventricular hypertrophy and complex phenotypic heterogeneity. While HCM is the most common inherited cardiomyopathy, pharmacological treatment options have previously been limited and were predominantly directed towards symptom control owing to left ventricular outflow obstruction. These therapies, including beta blockers, calcium channel blockers, and disopyramide, have not been shown to affect the natural history of the disease, which is of particular concern for younger patients who have an increased lifetime risk of experiencing arrhythmias, heart failure, and sudden cardiac death. Increased knowledge of the genetic mechanisms underlying this disease in recent years has led to the development of targeted, potentially disease-modifying therapies for both obstructive and nonobstructive phenotypes that may help to prevent or ameliorate left ventricular hypertrophy. In this review article, we will define the etiology and clinical phenotypes of HCM, summarize the conventional therapies for obstructive HCM, discuss the emerging targeted therapies as well as novel invasive approaches for obstructive HCM, describe the therapeutic advances for nonobstructive HCM, and outline the future directions for the treatment of HCM.
Packard et al. (Sat,) conducted a review in Hypertrophic cardiomyopathy (HCM). Targeted molecular therapies (e.g., cardiac myosin inhibitors) was evaluated. Emerging targeted therapies, including the newly FDA-approved cardiac myosin inhibitor mavacamten, provide novel disease-modifying treatment options for patients with hypertrophic cardiomyopathy.