A 35-year-old man presents with exertional dyspnoea and presyncope. Echo shows asymmetric septal hypertrophy (25 mm), systolic anterior motion of the mitral valve (SAM), and LVOT gradient of 65 mmHg at rest. He is on verapamil. Which drug is NEWLY FDA-approved (2022) for symptomatic obstructive HCM reducing LVOT gradient?
- A Mavacamten ✓
- B Metoprolol
- C Disopyramide
- D Ranolazine
Explanation
Mavacamten is a first-in-class cardiac myosin inhibitor that reduces excessive actin-myosin cross-bridge formation, thereby decreasing LVOT obstruction and SAM in obstructive HCM. The EXPLORER-HCM trial (NEJM 2020) showed significant reduction in LVOT gradient, improved exercise capacity, and NYHA class improvement. It received FDA approval in 2022. Metoprolol and verapamil are standard negative chronotropes/inotropes used in HCM. Disopyramide (negative inotrope) is used as an adjunct. Mavacamten's unique mechanism targeting the sarcomere distinguishes it as a disease-modifying therapy.
Reference: Harrison's Principles of Internal Medicine, 21st ed.
High-yield for: NEET PGINI-CETNExTFMGEUSMLEPLABMRCP
Written and medically reviewed by the StethoPrep medical team.