According to this recent article in the European Heart Journal, traditional cardiovascular risk factors such as obesity, hypertension and diabetes are associated with more severe forms of HCM. Therefore, the authors suggest that these additional risk factors should be aggressively managed so as to limit their impact on HCM.
A recent article on Biospace.com describes a method of targeted gene therapy for HCM patients currently being developed by Tenaya Therapeutics. This therapy is intended for patients whose HCM is caused by a mutation in the MYBPC3 gene.
According to Tenaya CEO Faraz Ali, animal models show that this therapy can reverse declining heart function.
You can find more information on about Tenaya’s approach here.
Arrhythmia monitoring in HCM patients is used not only for determining risk of sudden death and potential need for an implantable defibrillator, but also for detecting atrial fibrillation (AFib) and implementing anti-coagulation for stroke prevention.
A recent paper provides an overview of the usefulness of arrhythmia monitoring in patients with HCM.
According to this recent study looking at exercise capacity in patients with HCM, women with HCM demonstrated reduced exercise capacity when compared to men. This paper theorizes that the differences are likely attributable to passive diastolic properties and that these could aid in the development of interventions specifically targeted for women.
Cytokinetics has announced that its experimental drug aficamten, currently in trials as a potential treatment for obstructive hypertrophic cardiomyopathy, has received breakthrough therapy designation from the FDA. This designation is awarded by the FDA to certain drugs which may offer substantial improvement to patients over available therapies. The designation could shorten the FDA approval process for the drug by about 4 months.
You can read more about Cytokinetics and aficamten in these older posts from HCMBeat:
Interview with Dr. Martin Maron about Cytokinetic’s Drug Aficamten
Cytokinetics Announces Positive Results from REDWOOD-HCM Phase 2 Clinical Trial
Cytokinetics Moves Forward with HCM Drug Trial
Cytokinetics Announces its Phase 2 Clinical Trial
Positive Signs from REDWOOD-HCM
HCM Clinical Trials – the Latest News
2 Companies Testing Drugs for HCM
Scientists Get $10 Million Grant to Develop HCM Treatments
The U.S. Food and Drug Administration (FDA) has postponed the date by which it must complete its review of mavacamten – the first drug made specifically to treat HCM. The FDA was originally scheduled to announce its decision in late January 2022, but that date has now been postponed until April 28, 2022, which is the new “PDUFA date” or the date by which the FDA must respond to the new drug application.
The FDA has not asked Bristol Myers Squibb (BMS) to submit any additional data, but it wants to spend more time reviewing the proposed Risk Evaluation Mitigation Strategy or REMS which is the safety profile of the drug. The FDA can require REMS for certain drugs with safety concerns to ensure that the benefits of the medication will outweigh any risks.
BMS acquired the drug mavacamten, a first in class myosin inhibitor, through its $13.1 billion acquisition of San Francisco biotech company MyoKardia late last year.
You can read more about mavacamten and its journey in these past entries from HCMBeat:
2 Companies Testing Drugs for HCM
HCM Clinical Trials – the Latest News
HCM Drug Shows Improvement to Heart Structure
More Positive Data on Mavacamten Presented at ACC Meeting
More Positive Results for MyoKardia Drug
MyoKardia Announces Positive Results from EXPLORER Trial
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:
More data was presented by Bristol Myers Squibb (BMS) regarding its experimental drug mavacamten for obstructive hypertrophic cardiomyopathy. This data showed a sustained reduction in certain biomarkers of heart stress over a longer period of time. Additionally, BMS provided evidence that mavacamten maintains its effectiveness when taken in tandem with background beta blocker therapy. Read the complete abstract here.
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.
Berritt Haynes, a 19 year old with hypertrophic cardiomyopathy, was chosen for Team Blake Shelton last night on the MGM Television/NBC show THE VOICE. After Berritt’s mother submitted a tape, Berritt was chosen to audition on stage in front of the coaches.
Berritt had hoped to attend a taping of the show last season through Make-A -Wish Foundation which grants wishes to kids with serious health issues. However, due to COVID, he was not able to make that happen. Instead, this year his mother helped make his dream come true by making it possible for him to actually perform.
Berritt was diagnosed with HCM when he was 8 and received an implantable defibrillator when he was 14. Clearly, Berritt’s HCM has not interfered with his performing talents. Watch him performing on The Voice here.
Good luck Berritt. All of the HCM world will be rooting for you to advance to the next round!
Read more at:
THE VOICE can be seen on NBC on Monday and Tuesday nights. Check your local schedule for times.
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