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.
Continue reading “Mavacamten vs. Septal Reduction – VALOR-HCM Trial Results Published”
At long last, there is a FDA approved drug specifically intended for the treatment of hypertrophic cardiomyopathy. After several years of testing, and based on the results of the groundbreaking EXPLORER-HCM trial, Bristol Myers Squibb’s new drug mavacamten, being marketed under the brand name Camzyos, is now available to HCM patients.
Continue reading “FDA Approves Mavacamten under Brand Name Camzyos”
In addition to the presentation of the results of the VALOR-HCM study which compared mavacamten to septal reduction techniques, covered here in its own HCMBeat blog entry, ACC 2022 featured two other presentations about the new class of drugs known as myosin inhibitors, such as Bristol Myers Squibb’s mavacamten and Cytokinetics’ aficamten.
Continue reading “More on Aficamten & Mavacamten from ACC 2022”
The Phase 3 VALOR-HCM trial results were presented this morning at the American College of Cardiology annual meeting in Washington, DC by the principal investigator, Dr. Milind Desai of the Cleveland Clinic, and the results are good!
What was the VALOR-HCM study?
15 – 20 million people worldwide are estimated to have HCM, with 2/3 of this group having the obstructive form which can cause severe symptoms. Historically, these patients have been treated with medications approved for other conditions, and if those don’t relieve symptoms, they are referred on for septal reduction therapies (SRT) like alcohol septal ablation (a catheter based procedure) or septal myectomy (open heart surgery), which are invasive therapies requiring specialized care and which are not widely available.
The VALOR study was designed to compare mavacamten head to head with SRT to see if mavacamten could be a non-invasive treatment alternative for obstructive HCM.
Continue reading “VALOR-HCM Trial – Mavacamten vs. Septal Reduction Therapy – RESULTS ARE IN!”
Fifteen years ago, I referred myself to Mayo Clinic in Rochester, Minnesota, to be evaluated for a septal myectomy by what was at the time, one of the country’s few expert centers for the treatment of hypertrophic cardiomyopathy. I traveled to Mayo from my native state, Kentucky, on a brief hiatus from a well-established career in health policy in Washington, DC.
Continue reading “Guest Blogger Gwen Mayes, JD, MMSc – Cautiously Awaiting the Release of Mavacamten”
Bristol Myers Squibb has launched a new hypertrophic cardiomyopathy awareness campaign and website entitled “Could it be HCM?” The campaign launch is in connection with the expected early 2022 FDA approval for the first-in-class cardiac myosin inhibitor drug mavacamten,
A video made for the campaign features professional basketball player Jared Butler of the Utah Jazz. In the video, Butler shares his surprise and dismay when he learned of his HCM diagnosis. Butler was fortunate that he was cleared to play basketball by his doctors at the Mayo Clinic who continue to follow him closely. He was even featured in People Magazine talking about his HCM. See also this article in the Salt Lake Tribune.
The website described what happens to the heart in HCM, the symptoms of HCM, and provides resources for dealing with a diagnosis of HCM.
Check it out!
This year at the AHA Scientific Sessions several presentations focused on HCM. A couple receiving attention included:
On October 22, patients, physicians, and other interested parties will have the opportunity to provide input on the value and cost of mavacamten – the first drug specifically designed to treat hypertrophic cardiomyopathy. Last year, Bristol Myers Squibb paid $13.1 billion to acquire MyoKardia, the San Francisco biotech company that developed the drug and brought it through clinical trials.
At a virtual public meeting, The Institute for Clinical and Economic Review or ICER will listen to further testimony in order to evaluate mavacamten’s value and potential benefits. ICER is a non-profit organization that evaluates the cost effectiveness of drugs and medical procedures. Many insurance companies rely on ICER’s findings when deciding how much to pay for a certain treatment or test.
In an Effectiveness Report which was published today, ICER valued the benefit that mavacamten would bring to a patient at between $12,000 to $15,000 a year. By contrast, some analysts have suggested that mavacamten could carry a price tag as high as $75,000 per patient per year.
If you would like to share your thoughts at the online public meeting click here to sign up.
You can find a press release from ICER about their review of mavacamten here.
Editor’s note: You have probably noticed a distinct uptick in clinical trials of potential treatments for hypertrophic cardiomyopathy. HCMBeat has been following this trend and has previously published a host of stories about such trials, including this story about the positive results from the REDWOOD-HCM Phase 2 clinical trial, as well as past stories discussing the biopharmaceutical company Cytokinetics, its drug aficamten (previously known as CK-274), and the REDWOOD-HCM trial.
Some of these earlier stories are as follows:
2 Companies Testing Drugs for HCM
HCM Clinical Trials – the Latest News
Positive Signs from REDWOOD-HCM
Cytokinetics Moves Forward with HCM Drug Trial
Recently, Cynthia Waldman of HCMBeat had the opportunity to speak over Zoom with Dr. Martin Maron, who recently served as the principal investigator of Cytokinetics’ REDWOOD trial. The conversation focused both on Cytokinetic’s drug aficamten (previously known as CK-274), and the new class of drugs known as “myosin inhibitors.” What follows is a transcript of their conversation (which has been edited for readability).
Continue reading “Cytokinetic’s Drug Aficamten & Upcoming HCM Summit – Interview with Dr. Martin Maron”