Novel drug proving beneficial in HCM patients

Patients with obstructive hypertrophic cardiomyopathy (HCM) are showing sustained benefits from the novel drug mavacamten. Results from the EXPLORER-HCM extension study (the MAVA-LTE trial) found patients using the drug had significant improvements in their health status, symptoms, exercise capacity and quality of life compared to similar patients using placebo after 30 weeks. “Mavacamten is the first medication specifically designed to relieve this obstruction . . .. If this medication continues to be effective over time, it may also help keep patients from having to undergo an invasive procedure or open-heart surgery to relieve the obstruction,” according to researchers from Cedars Sinai who presented their findings at a late-breaking session at the recent American College of Cardiology conference. MAVA-LTE was an ongoing, dose-blinded five-year extension study that included 231 of 244 patients who completed treatment in the phase-3 EXPLORER-HCM trial. This extension trial was designed to collect longer-term data. Data were collected from 19 sites across the U.S. Read: Which anticoagulant is the best to prevent recurrent VTE? Patients initially were given 5 mg of once daily mavacamten. Later they were given 2.5, 5, 10 or 15 mg, with doses adjusted at weeks four, eight and 12 and again at 24 weeks. The doses used varied according to the degree of obstruction by gradient and ejection fraction measures. Median follow-up was 62 weeks (up to 124 weeks). According to an ACC report, at baseline, 94% of patients had some degree of shortness of breath with exercise compared with only 45% at 48 weeks. At follow-up, the degree of obstruction as measured by left ventricular outflow tract gradients was lowered by an average of 36 mmHg at 48 weeks—a 74% reduction from baseline and was sustained through week 84 in those with longer follow-up. Overall, 68% of patients improved by at least one class designation on the New York Heart Association (NYHA) Functional Classification. The report stated the most dramatic change was in Class I, “with the percentage of patients at Class 1 jumping from only 6% at baseline, to 55% by week 48. In contrast, 29% of patients were in Class III, showing noticeable limitation with physical activity at the start of the study. The proportion of people in this class dropped to 4.9% at 48 weeks.” An earlier study found that the drug was associated with favourable cardiac remodeling in HCM patients. Mavacamten is under review with the U.S. Food and Drug Administration and Health Canada. Read: Aspirin beneficial to hospitalized COVID-19 patients

Novel drug proving beneficial in HCM patients

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