The use of mavacamten, a cardiac myosin inhibitor, for patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM), has shown continuous and consistent benefits in quality of life and long-term outcomes, according to interim results of the EXPLORER study HCM.
These late-breaking findings were presented at the American College of Cardiology (ACC) 2022 Scientific Sessions in Washington, DC.
In MAVA-LTE, the drug was associated with significant improvements in left ventricular outflow tract (LVOT) gradients, as well as improvements in both NT-proBNP and NYHA class.
In an interview with HCPLivelead author Florian Rader, managing director, co-director, clinic for hypertrophic cardiomyopathy and orthopedics, Cedars-Sinai, highlighted the importance of more data on the new medication class for HCM.
“What people were looking for, I think, is to get more long-term safety data from this totally new class of medication,” Rader said. “And we were happy to find that there were no new safety signals, no new concerns, there were a few cases of patients dropping their ejection fraction below the 50% protocol-defined cut-off point. All of these patients, the reduction in ejection fraction, all of which were reversible. “
He noted the importance of long-term expansion in determining that long-term exposure to the new drug does not cause increased incidence of low ejection fractions.
“Overall, I think these results are very encouraging that it will not happen,” Rader said. “But we will follow those patients and make sure they are doing well over the entire five-year follow-up period.”
Rader also expressed his thoughts on mavacamten as a game changer for patients with HCM and prevents them from having to undergo an invasive procedure.
“Since HCM was first described, more than 60 years ago, there really has not been any specific targeted drug therapy for this disease and mavacamten is now really the most promising candidate for this role.”
Aware of his potential approval at the end of April, Rader discussed how the survey could be for the agent.
“I think as with any new medication class, there are likely to be larger HCM centers that are likely to adopt this new medication a little faster than smaller practices,” he said. “And it will also likely depend on how strict the safety monitoring requirements will be for this new medication and price may also play a role.”
“Updated cumulative results of Mavacamten treatment from the EXPLORER-LTE cohort of the MAVA-LTE study in patients with obstructive hypertrophic cardiomyopathy,” were presented at ACC 2022.