FDA Approval of Mavacamten Delayed

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

MyoKardia’s EXPLORER Trial Big Success

Mavacamten Approaches Finish Line

BMS Launches New HCM Awareness Campaign Featuring Utah Jazz Player Jared Butler

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!

HCM News from 2021 AHA Scientific Sessions

This year at the AHA Scientific Sessions several presentations focused on HCM.  A couple receiving attention included:

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”

ACC Focuses on HCM with Cover Story

The July edition of the the American College of Cardiology’s magazine features a cover story about HCM. 

The article surveys the history of HCM and then moves through the evolution into contemporary treatments.  The article also contains a summary of important topics from the new 2020 AHA/ACC Guidelines and highlights EXPLORER-HCM, the recent groundbreaking clinical trial of mavacamten

Check it out when you can.

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

 

Experts Put Mavacamten in Perspective

An article published today in Circulation by HCM experts Dr. Steve Ommen of Mayo Clinic and Dr. Martin Maron of Tufts Medical Center, discusses the prospective use of mavacamten as a treatment for obstructive hypertrophic cardiomyopathy. The doctors conclude that while mavacamten (assuming that it is FDA approved in early 2022) will have its place in the HCM tool kit, it should not replace septal reduction therapy for severe HOCM.

In particular, the article points out that the EXPLORER-HCM study showed modest improvements in symptoms and functional capacity (peak V02), comparable to those seen in the RESET-HCM study, which highlighted the ability of regular exercise to improve functional status in HCM.  

The article notes that there has not yet been a study directly comparing mavacamten with septal reduction therapies such as septal myectomy and alcohol septal reduction. The VALOR-HCM study, which is currently recruiting, will look at these therapies compared head-to-head.  It is noteworthy that the majority of patients in the EXPLORER trial had Class II heart failure and were not the more severely compromised Class III and IV patients most likely to benefit from myectomy or alcohol septal ablation.

This article compared historical myectomy data against the findings from EXPLORER, concluding that septal myectomy produces a better result for patients, with gradients abolished in more than 95% of patients compared to only 50% of patients with mavacamten.  And, the article points out that 25% of the patients in the EXPLORER trial continued to have left ventricular outflow tract gradients greater than or equal to 50mmHg, which still qualified them for septal reduction therapy.

Maron and Ommen’s take-home message is that mavacamten will be a welcome addition to the arsenal of HCM drugs and is potentially suitable for patients who do not have severe symptoms, who do not have access to septal reduction at a HCM specialty center, or who wish to avoid more invasive therapies.  It also may be used in the same way as disopyramide, to defer surgery by improving symptoms to a tolerable level.

Lastly, this article points out that there is a need for longer term follow up to study the effects of cardiac remodeling caused by mavacamten.

While it is wonderful to have options, it is important that patients and their medical team consider all available information, including potential benefit and risk, before moving forward with medical therapy. 

Mavacamten Approaches Finish Line

The United States Food and Drug Administration (FDA) is expected to complete its review of mavacamten and release its decision on whether to approve the drug for sale in the U.S. by January 28, 2022.

This week, Bristol Myers Squibb submitted its New Drug Application for mavacamten to the FDA. Mavacamten is the first drug specifically designed to treat obstructive hypertrophic cardiomyopathy. The application was based on the recent positive results of the Phase 3  EXPLORER-HCM trial

In October of last year, Bristol Myers Squibb paid $13.1 billion to purchase MyoKardia, the San Francisco based biotech company which developed mavacamten as a novel cardiac myosin modulator for the treatment of of HCM.

The FDA has assigned a Prescription Drug User Fee Act goal date of January 28, 2022 to the drug, which means that the FDA is expected to complete its review of mavacamten by January 28, 2022.

 

 

 

 

 

 

DISCLOSURES:  CYNTHIA BURSTEIN WALDMAN OF HCMBEAT SERVED AS A PATIENT ADVISOR ON THE STEERING COMMITTEE OF MYOKARDIA’S EXPLORER TRIAL AND IS AN AUTHOR OF THE STUDY AS PUBLISHED IN THE LANCET.  CYNTHIA ALSO SERVES ON MYOKARDIA’S PATIENT ADVISORY BOARD.

 

HCM Drug Shows Improvement to Heart Structure

The results from the Myokardia EXPLORER- MRI sub-study are in, and there is more positive news for mavacamten (formerly known as MYK-461). According to the results of this small 35 patient study, patients who took mavacamten showed reduction of left ventricular size and wall thickness on MRI. These patients also had a reduction in their left atrial volume. All three of these measurements are predictors of poor outcome for HCM patients Additionally, this study found a reduction in certain biomarkers such as NT-proBNP, which indicate heart stress and injury.

MyoKardia (which was recently acquired by Bristol Myers Squibb) hopes that this drug will be approved by the FDA and available to American HCM patients by the end of this year.

For more on the progress of mavacamten, read these previous entries on HCMBeat:

Positive Results for MyoKardia Drug Mavacamten

MyoKardia Announces Positive Result for Mavacamten for Treatment of HOCM

MyoKardia Announces Positive Results from EXPLORER Trial

More Positive Results for MyoKardia Drug

MyoKardia’s EXPLORER Trial Big Success

 
 
 
DISCLOSURES:   CYNTHIA BURSTEIN WALDMAN OF HCMBEAT SERVED AS A PATIENT ADVISOR ON THE STEERING COMMITTEE OF MYOKARDIA’S EXPLORER TRIAL. CYNTHIA ALSO SERVES ON MYOKARDIA’S PATIENT ADVISORY BOARD