The Mavacamten Marathon

Camzyos™ Approval

This is an exciting time for the HCM Community.  The FDA has approved Mavacamten under the brand name CAMZYOS, the first-in-class medication for symptomatic obstructive hypertrophic cardiomyopathy and the first drug developed specifically for HCM.

Read the press release here

CAMZYOS Patient Portal

CAMZYOS Medication Guide

CAMZYOS U.S. Prescribing Information

CAMZYOSTM REMS (Risk Evaluation and Mitigation Strategy)

Our website will provide as much information as possible as it is released by BMS to be a trusted source of information during this exciting time. For the latest information please click here.

In June, there will be a webinar with more information forthcoming discussing in detail the label, the patients who may benefit, drug interactions, how to access the patient assistance programs, and more.    

This is a historic day for our community, and it is important to take a moment and take a collective deep breath and admire the view.  

The approval notification from the FDA is as unique as HCM, as it has specific language that is intended to ensure patient safety by the requirement of serial echocardiography to be used in dosing and observation.  We believe it to be the first-ever such requirement and we applaud both the FDA and BMS for this language.

While this is the finish line, it is only the beginning of the true journey of a deeper understanding of the utility of this novel medication.  

We are simultaneously at a finish line and a starting line. About 8 and a half years ago the HCMA met with a small startup company with plans of bringing a new class of medication specifically to the HCM Community. 

The concepts and the science sounded strong, however, there was a long process of development, safety testing, therapeutic testing, analysis, filing for approval, supply chain processes, provider education, payer education, patient education, and finally –  a ruling from the FDA.   

There is a very special group of people we must stand and applaud – those who participated in clinical trials – every single patient who answered a survey, attended an education session, spoke to a clinical trial coordinator, signed up, and completed the trial. It is your time, energy, bravery, commitment, and passion that brought us to this historic moment. 

It did “take a village” to get us here so let us also acknowledge all stakeholders in this process, this starts with venture capital firms who provided the needed capital for a startup operation called MyoKardia to begin its work.  The staff at every level of MyoKardia set the course – I include senior leadership, scientists, marketing, and contractors as well as the clinical investigator team which encompasses many of our HCMA recognized Center of Excellence partners.  

In 2020 as the Covid-19 pandemic was unfolding, the HCMA held an externally led Patient-Focused Drug Development Meeting inviting the FDA to hear directly from HCM patients about the true burden of HCM in the lives of those with HCM.   

Then about a year and a half ago, Bristol Myers Squibb purchased MyoKardia and this provided a completely different team stepping in to take Mavacamten to the “finish line” and beyond.  

The new BMS team came in with a limited understanding of HCM and I am pleased to say that communications with the HCMA began within days of the BMS acquisition. Many long conversations took place to ensure our unique community was understood at the deepest level possible.  

The efforts of Bristol Meyers Squibb to educate clinicians about HCM have been nothing short of extraordinary and the HCMA is a proud partner in nearly a dozen various educational programs with partners from AHA, ACC, WomanHeart, Global Heart Hub, PCM Scientific, Prime, Peerview, and many others.  

While the VALOR, Explorer, and the long-term extension trials are complete and we have a label for those with symptomatic obstructive HCM (typically referred to as HOCM) we are all still learning together about the optimal patients for this new “tool” in the HCM “Toolbox”.   We believe it will be a beneficial option for some but caution that it will not work for everyone. The HCMA wants to be clear in our communications that this is not a cure for HCM. It is in fact a targeted therapy that some will find very helpful in managing HCM.   

This is why I say we are at the starting line.   It is time to understand the real-world use of Mavacamten specifically and myosin inhibitors in general.  We encourage those with obstruction to discuss this treatment option with your HCM Center of Excellence care team to determine if it is right for you.  

Over the years there have been high hopes for particular therapies, DDD Pacing in the early 90s, Alcohol septal ablation in the early 2000s, and other novel agents that never made it to the finish line.  We learned that each of these concepts was not THE answer for all, but an option for some.   I believe it is time to celebrate new options for those in need of hope and improved quality of life. 

We know you have many questions and the HCMA will be providing support during these changing times.  This will include a new player in your healthcare team, a specialty pharmacy. Mavacamten will only be available from a unique class of distribution reserved for medications that are both expensive and need specific monitoring to prescribe.  Your insurance programs will guide you to the specialty pharmacy number. 

The price of Camzyos is likely to evoke a bit of sticker shock at $90,000 per year, however, there are patient assistance programs with the end price being under $20.00 for the majority of those in the USA.  The HCMA has worked very closely with BMS to deliver the most expansive patient assistance program in the cardiovascular space including both medication cost assistance and echocardiogram payment assistance (first ever!).  

A special message to those outside the USA – additional countries will be seeking authorization of Mavacamten in the coming months or years, and it is anticipated that by 2024 it may be available globally. 

My sincere thanks to all who have been a part of this important chapter of HCM history.  I end with a word of balance and cautious optimism. We will learn together the effectiveness, sustainability, and role of myosin inhibitors for the HCM community.  On your marks, get set, go – cautiously into the future together. 


Lisa Salberg

Founder and CEO

Get evaluated by an HCMA recognized Center of Excellence to see if Camzyos (Mavacamten) is right for you. Camzyos is not available in local pharmacies.

Click here to see the list our list of Centers of Excellence.