WASHINGTON – Treatment of patients with symptomatic obstructive hypertrophic cardiomyopathy who remained on treatment with the investigational agent mavacamten for a median of 62 weeks continued to show the same level of safe response to the drug as seen after the first 30 weeks on treatment in the pivotal trial for this agent.
The new findings from longer-term treatment bode well for mavacamten. That’s because if the drug is used in routine practice to avoid the need for surgery or an invasive intervention to reduce blockage of a patient’s left ventricular outflow tract, the duration of mavacamten treatment will likely need to continue for many years and even for decades, said Florian Rader MD , who presented the results at the annual scientific sessions of the American College of Cardiology.
In practice, mavacamten will probably be used for many, many years, especially as it replaces septal-reduction therapy, so we need long-term data,” noted Dr. Rader during a press briefing on his report. “I’m very happy with the long-term data” in the follow-up study.
The Food and Drug Administration is currently considering whether to approve mavacamten for routine marketing to treat patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM), with a Decision expected by the end of April 2022.
The study Dr. Rader reported followed 231 patients with symptomatic oHCM who had completed the 30-week pivotal trial of mavacamten, EXPLORER HCM , and opted to continue on open-label extended treatment with mavacamten, either continuing the treatment they started during the trial or crossing over to receive mavacamten after receiving placebo during the trial.
The major findings from EXPLORER – LTE (long-term extension) were that continued treatment for a median of about 62 weeks maintained the safety and efficacy findings seen at the end of the blinded, randomized, initial 30 week Phase , said Dr. Rader, codirector of the Clinic for Hypertrophic Cardiomyopathy and Aortopathies at Cedars-Sinai Medical Center in Los Angeles.
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