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

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

 

 

What Should Mavacamten Cost?

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 benefitsICER 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.

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”

Drug Trial for Non-Obstructive HCM

Yet another company is developing a new drug for hypertrophic cardiomyopathy, and this time, the drug is intended for non-obstructive HCM patients.  The company is Imbria Pharmaceuticals, and this week they announced the randomizing of the first patient in their Phase 2 study of the drug IMB-101 in patients with non-obstructive HCM.  The study, called IMPROVE-HCM, is a Phase 2 study that will look at the safety and tolerability of this drug in non-obstructed HCM patients. IMB-101 is designed to increase the efficiency of the heart’s use of energy which will be measured through cardiopulmonary exercise testing over a 12 week period.

You can read this press release and you can read more about the trial on ClinicalTrials.gov here.

More Positive Data on Mavacamten Presented at ACC Meeting

More positive data about the Bristol Myers Squibb experimental drug mavacamten was revealed at last weekend’s American College of Cardiology meeting and simultaneously published in The Lancet.

The data showed improvement in how patients felt taking the drug, as reported and quantified by the patients themselves. The clinical trial participants filled out a questionnaire called the KCCQ, or Kansas City Cardiomyopathy Questionnaire, 6 different times over the 38 weeks that the trial was ongoing.

Continue reading “More Positive Data on Mavacamten Presented at ACC Meeting”

Cytokinetics Announces its Phase 2 Clinical Trial – “Redwood-HCM”

Cytokinetics today announced that its Phase 2 double-blind study of its experimental drug CK-274 entitled “REDWOOD-HCM” (Randomized Evaluation of Dosing With CK-274 in Obstructive Outflow Disease in HCM (hypertrophic cardiomyopathy) has begun enrollment.  The trial will enroll patients with symptomatic, obstructive HCM.

CK-274 is a next-generation cardiac myosin inhibitor which the company hopes will prove to be beneficial for the treatment of hypertrophic cardiomyopathy (HCM).

There are currently two companies in clinical trials for HCM:  Cytokinetics and MyoKardia. You can read more about their efforts here and here.

 

2 Companies Testing Drugs for HCM

Two San Francisco based companies are now conducting clinical trials for three drugs specifically targeting HCM.

Continue reading “2 Companies Testing Drugs for HCM”

Scientists Get $10 Million Grant to Develop HCM Treatments

A group of scientists led by Stanford University’s Dr. James Spudich, working together with researchers from the University of California-Santa Barbara, the University of Washington and the Institut Curie in Paris, has recently been awarded a $10 million grant by the National Institute of General Medical Sciences to develop novel treatments for hypertrophic cardiomyopathy (HCM). 

The researchers hope that the added resources from this grant will help them find ways to correct pathological heart protein changes they believe to be at the root of HCM. The team then plans to partner with pharmaceutical companies to develop more personalized approaches to HCM treatment.

Dr. Spudich has long been involved in HCM research and has been a founder of two separate companies which are currently engaged in drug trials for potential HCM treatments:  MyoKardia and Cytokinetics.

A story about Dr. Spudich and the inspiration for his work was featured in this recent post on HCMBeat.