The VALOR-HCM trial results have just been published in the Journal of the American College of Cardiology. 

This study enrolled 112 obstructive HCM patients for a 16 week double blind trial of the drug mavacamten (brand name Camzyos). All patients in the trial had been referred for septal reduction therapy – either septal myectomy or septal alcohol ablation – to treat their highly symptomatic obstructive hypertrophic cardiomyopathy.   The researchers looked at whether the addition of mavacamten to their other drugs would improve their symptoms enough so that they no longer met the criteria for septal reduction therapy (SRT) under the 2011 ACC/AHA Guidelines.

You can read many more details about the VALOR-HCM study here in this recent blog post on HCMBeat.

The bottom line, however, is that following the trial, 82% of the patients enrolled no longer qualified for SRT, while 18% or 4 patients decided to continue on to surgery and/or met the guidelines for SRT eligibility.  And, 95% of patients in the study chose to continue on with mavacamten as part of a longer term study. 

An accompanying editorial comment by Dr. Steve Ommen of Mayo Clinic puts mavacamten into context by looking at it alongside other more established HCM treatments.

Dr. Ommen says that the true benefits of mavacamten will not be known until doctors have had an opportunity to see how it works in the real world over a much longer period of time.

And, there are complexities that patients need to consider when using mavacamten which are not present with older drugs used to treat HCM. The FDA requires a boxed warning and a Risk Evaluation and Mitigation Strategy (REMS). The medication can only be dispensed by specialty pharmacies while patients must undergo at least 7 evaluations which include echocardiograms during the first year of therapy. Following the first year, 4 evaluations will be required yearly as long as a patient stays on the drug.

Before choosing any particular pathway, patients should be advised of risks, benefits, logistics and costs of each intervention. Then, the patients, with the guidance of their physicians, can make the best decision for themselves based on their own circumstances.

Some will choose mavacamten to delay surgery, while others will hope that long term therapy will obviate the need for surgery or ablation.  Others may prefer to undergo a more definitive intervention like myectomy. And there are other factors such as cost or insurance which may dictate choices.

In any event, as Dr. Ommen says, “time will tell.”