A 35-year-old man has hypertrophic obstructive cardiomyopathy (HOCM) with peak left ventricular outflow tract (LVOT) gradient 68 mmHg at rest, NYHA III symptoms despite maximum tolerated beta-blocker. What is the newer pharmacological agent targeting the cardiac sarcomere that is now approved for symptomatic HOCM?
- A Ranolazine (late sodium channel inhibitor)
- B Ivabradine (If channel inhibitor)
- C Mavacamten (cardiac myosin inhibitor) ✓
- D Empagliflozin (SGLT2 inhibitor)
Explanation
Mavacamten is a first-in-class cardiac myosin inhibitor that reduces excessive actin-myosin cross-bridge formation, the fundamental mechanism causing hypercontractility and LVOT obstruction in HCM. The EXPLORER-HCM trial demonstrated significant reduction in LVOT gradient, improvement in NYHA class and exercise capacity, and reduction in septal reduction therapy (SRT) eligibility. FDA and EMA approved mavacamten in 2022 for symptomatic obstructive HCM (HOCM) as an alternative to invasive SRT (septal myectomy or alcohol septal ablation). It requires REMS programme due to HFrEF risk (LVEF monitoring required).
Reference: Harrison's Principles of Internal Medicine, 21st ed.
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Written and medically reviewed by the StethoPrep medical team.