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

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