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.

More on Aficamten & Mavacamten from ACC 2022

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”

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

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!”

Traditional Cardiovascular Risk Factors Worsen HCM

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.  

Gene Therapy for 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

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. 

Continue reading “Arrhythmia Monitoring in HCM”

Worse Exercise Capacity in Women 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.

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”

Aficamten Gets “Breakthrough Drug” Status from FDA

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

The Future of HCM Care

HCM Clinical Trials – the Latest News

2 Companies Testing Drugs for HCM

Scientists Get $10 Million Grant to Develop HCM Treatments