FDA Approves Mavacamten under Brand Name Camzyos

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.

However, you will not be able to go to your regular drug store and obtain a prescription of Camzyos: the drug is subject to a black box warning applicable to certain medications that have the potential to cause serious harm to the user. Camzyos will only be prescribed through a restricted program called the Camzyos Risk Evaluation and Mitigation Strategy (REMS).  The drug will also be sold only by certain specialty pharmacies which understand how the drug works and the risks that it can present. Also, there will be a screening for drug interactions as Camzyos may interact with other drugs you may be taking.  Lastly, patients who are prescribed Camzyos will be required to undergo echocardiograms every 4 weeks for the first three months of treatment, and then every three months thereafter to ensure that the drug hasn’t moved the patient into heart failure.

Even with these restrictions and limitations, this is exciting news for patients with HCM. Hopefully the coming years will show increased safety data and expanded indications for Camzyos.  And, we hope that the list price which is approximately $89,000 annually will not prove to be prohibitive.  Bristol Myers Squibb has put into place a Patient Support program which will hopefully help make the drug and the necessary medical follow up affordable for most patients.

Here is the official Camzyos website for patients.

Here is the official Camzyos website for physicians.

Here is the REMS patient brochure that explains what kind of monitoring is necessary for patients to undergo and what conditions the patient must agree to in order to receive the medication.

Here is what is required to become a certified specialty pharmacy dispensing Camzyos.

And here are some past HCMBeat blog posts about mavacamten from the early days of the South San Francisco biotech company MyoKardia until today. As you can see, there is a lot to catch up on!

MyoKardia HCM Drug Has Success in Cats

Encouraging Results for MyoKardia HCM Drug

MyoKardia Drug Moves to Next Phase

Encouraging Results for MyoKardia HCM Drug

MyoKardia Drug Moves to Next Phase

MyoKardia Announces Positive Result for Mavacamten for Treatment of HOCM

Positive Results for MyoKardia Drug Mavacamten

More Positive Results for MyoKardia Drug

MyoKardia Announces Positive Results from EXPLORER Trial

MyoKardia’s EXPLORER Trial Big Success

MyoKardia Acquired by Bristol Myers Squibb – UPDATED

HCM News from 2021 AHA Scientific Sessions

Mavacamten: A Plain Language Summary of EXPLORER – HCM

What Should Mavacamten Cost?

Guest Blogger Gwen Mayes, JD, MMSc – Cautiously Awaiting the Release of Mavacamten

VALOR-HCM Trial – Mavacamten vs. Septal Reduction Therapy – RESULTS ARE IN!

More on Aficamten & Mavacamten from ACC 2022

Sports and HCM – Moving Toward Shared Decision Making

While competitive sports used to be frowned upon in the HCM literature, there is now some evidence that a patient’s risk from exercise is low when they have been implanted with an implantable cardioverter defibrillator (ICD). This feature from Medpage Today gives an overview of contemporary thinking about exercise in HCM patients.

Comprehensive risk assessment, combined with shared decision making around the decision to participate in sports, seems to be the way of the future. This is more fully discussed in the most recent ACC/AHA Guidelines.

However, ICDs should only be implanted if the patient is clearly at risk of sudden cardiac arrest;  they should not be implanted solely for the purpose of allowing a low risk patient to participate in sports.

Guest Blogger Gwen Mayes, JD, MMSc – Cautiously Awaiting the Release of Mavacamten

            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”

Cytokinetic’s Drug Aficamten & Upcoming HCM Summit – Interview with Dr. Martin Maron

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”

Fifteen Year Anniversary of Myectomy!

Today it has been 15 years since my septal myectomy at Mayo Clinic!  

 
It’s hard to believe, and lots has happened since my open heart surgery, but I wanted to post today to let everyone know that it was totally worth it. The best decision I ever made.  I have been working full time since the surgery, have been working on this blog and have a busy life as a wife, mother and daughter. 
 
 
Of course the COVID pandemic has been challenging for all of us, but I am grateful for the blessing of the great medical care that has allowed me to continue living my best possible life, even if it is currently limited by the circumstances.
 
If you are on the fence wondering if this surgery is worth it, I am here to tell you it absolutely was.  You can see a collection of resources I gathered about myectomy.  Also, if you are wondering what it is like to visit a place like Mayo Clinic, I wrote a travelogue here.
 
And here are some words from my Mayo Clinic cardiologist, Dr. Steve Ommen, about the role of a specialty center in HCM care. Or you can read a recent interview with Dr. Ommen on Medscape here.
 
Meanwhile, wishing you all good health!

Mavacamten: A Plain Language Summary of EXPLORER – HCM

I was recently asked by the good people at MyoKardia, now a subsidiary of Bristol Myers Squibb, if I would author a plain language summary of an article to be published in the journal Future Cardiology.  This article summarizes the results of the EXPLORER-HCM trial, which showed that mavacamten, a new type of drug known as a “myosin modulator,” was successful in reducing HCM symptoms in subset of patients who took the drug during the clinical trial. 

I was so fortunate to have Dr. Anjali Owens of the University of Pennsylvania Center for Inherited Cardiac Disease join me as co-author on the article.  I also want to thank the teams at Bristol Myers Squibb and Cello Health Communications/SciFluent who supported us on this project.  And, I so grateful to the former MyoKardia team and the other members of the EXPLORER-HCM Steering Committee who gave me the opportunity to witness a groundbreaking clinical trial from the front row.

This is the article.  When you access the page, click to view the very informative short video, located in the upper right corner of the page, which explains how the drug works in very brief and concise terms.

Check it out!

Cynthia Burstein Waldman, Founder and Editor, HCMBeat

 

When the Doctor Becomes a Patient

Just about everyone involved with HCM has heard the name Dr. Barry J. Maron.  Dr. Maron has devoted his entire career to hypertrophic cardiomyopathy and has been perhaps the physician most associated with HCM, having gotten his start in the 1970s at the National Heart Institute.

In a brand new autobiographical essay by Dr. Maron, he retells his recent experience being diagnosed and treated for heart failure. 

When Dr. Maron experienced edema, difficulty lying flat, and trouble breathing, he rationalized it all away until he experienced a terrifying and life threatening episode, and even then, he decided to call an Uber to take him to the hospital instead of an ambulance! 

Once hospitalized, doctors were able to get to the root of his problem and treat Dr. Maron appropriately. Having received the proper care, he is now feeling much better.

Dr. Maron’s article concludes with three main takeaways:

  • Physicians should not diagnose themselves;
  • Heart failure is treatable; and
  • Listen to your cardiologist and live a healthy lifestyle!

AHA Seeking Patient Input for New HCM Initiative

In November at the American Heart Association’s Scientific Sessions held in Philadelphia, AHA announced that it would be starting a three-year initiative focused on hypertrophic cardiomyopathy, also known as HCM.  The initiative is sponsored by MyoKardia, a San Francisco based company that is currently in clinical trials for mavacamten, the first drug specifically intended to treat HCM.

Last week, Cynthia Waldman of HCMBeat had the opportunity to speak with Amy Schmitz, AHA’s National Corporate Relations Director and Alexson Calahan, a Communication Manager for AHA.

What follows is a summary of their conversation about the forthcoming HCM initiative that has been edited for clarity.

Continue reading “AHA Seeking Patient Input for New HCM Initiative”

MRIs for ICD and Pacemaker Patients

Here is an informative new video from our friend Doug Rachac that nicely explains the safety of MRIs for patients with implantable defibrillators and pacemakers.

I wrote a blog piece about this same issue a few years back. Here it is:

Yes We Scan! ICDs and MRIs

And a few other relevant blog entries here on HCMBeat:

Study Shows MRIs Safe for Pacemaker & ICD Patients

Chapter 3: MRI Safety for ICD & Pacemaker Patients

Safety of MRIs With Abandoned Leads

Last year, Doug wrote this blog entry for HCMBeat specifically about magnets and airports. Read that here:

Blogger Doug Rachac – Magnets and Airports: Should ICD Patients Be Afraid?

And, you can find more about ICDs from Doug on his YouTube Channel.

Dr. Harry Lever Speaks Out About Problems With Generic Drugs

This Medscape article highlights the extraordinary efforts of Dr. Harry Lever, Director of the Cleveland Clinic’s Hypertrophic Cardiomyopathy Center, in educating patients and physicians alike about quality issues with generic drugs.  Dr. Lever has been instrumental in publicizing the fact that generic drugs are NOT always the same as their name brand counterparts, and that foreign generics are not put through the same level of scrutiny as drugs in the U.S.

Continue reading “Dr. Harry Lever Speaks Out About Problems With Generic Drugs”