Experience With A New Miracle Drug " Mavacamten" For Diasbled Patients With Hypertrophic Obstructive Cardiomyopathy (HOCM)HCM is one the commonest cardiomyopathies but often remains undiagnosed. It is estimated to occur in 1 out of 200 to 500 subjects and 80-90% remain undiagnosed. It has 2 varieties, HCM with obstruction (HOCM) and HCM without obstruction (Non-Obstructive HCM). The basic pathology is dysfunction of the sarcomeres leading to excessive actin myosin cross bridging. This leads to excessive contractility, impaired relaxation and poor compliance. This leads to stiffness of the heart and eventual fibrosis. It is a chronic progressive myocardial disease. Diagnosis can be suspected by an ECG a clinical examination. It is finally confirmed by echocardiography and doppler studies which also differentiate between HOCM and NOHCM a provocative test like Valsalva maneuver is very important. A gradient across the left ventricular outflow which is often labile of more than 30 mms Hg is diagnostic. Untreated patients can get heart failure (20%- 45%), arrhythmias in 4% -45% (atrial fibrillation, non-sustained ventricular tachycardia), stroke and rarely sudden cardiac death. Most patients with HOCM are very symptomatic with shortness of breath, palpitations, fainting.
Disease often runs in families. Traditional treatment has been with beta-blockers like metoprolol in high doses, heart lowering calcium blockers like verapamil and non-specific agents like di-disopyramide. These drugs are only of marginal help with very little effect on obstructive gradients. Cardiac surgery and procedures like septal reduction by blocking the arteries supplying have been performed in very symptomatic patients. Recently cardiac myosin inhibitors, like Mavacamten which target the overactive heart muscle protein (myosin) to reduce obstruction, improve symptoms, and exercise capacity in adults with obstructive HCM, offering an alternative to surgery Mavacamten was first released in USA in April 2022 and in Europe in June 2023. It was released in India on 13th October 2025.
My group has used it so far in 11 patients . The results have been phenomenal with a near abolishment of the obstruction even with provocation with normalization of the hypertrophied muscle like a miracle. These patients have after several years of suffering started leading a normal life. The therapy has to be long term. The product is from the well-known company Bristol Myers Squibb. A case study with Echo-Doppler studies before and after 3 weeks of Mavacamten is attached.
Another product working on the same principal Aficamten which also offers reversibility and potential for nonobstructive HCM use too has been released last month on 19th December. It is produced and marketed by Cytokinetics a USA based pharmaceutical company.
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