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.
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.
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.
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:
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:
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.
Check it out!
This year at the AHA Scientific Sessions several presentations focused on HCM. A couple receiving attention included:
- SPIRIT-HCM, a Cleveland Clinic study which looked at the effectiveness of myectomy in improving symptoms in patients with obstructive hypertrophic cardiomyopathy. This study used patient self-reported questionnaires to measure each patient’s improvement in symptoms and quality of life following surgery. The study showed that myectomy is highly effective in reducing symptoms and in improving quality of life following surgery.
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.