Gordon Fox • March 10, 2025
Camzyos is Safe

REMS works to find, stop, heart failure

The new drug Camzyos (mavacamten) has been nothing less than life changing for some patients who have HCM with obstruction. But like any drug, not all patients can take it, and not all those who take it benefit enough to stay with it. It can also add complication to a patient's life. Insurance coverage is often unpredictable. The FDA-mandated REMS  (Risk Evaluation and Management System) and warnings may seem daunting to some.

REMS is a good thing

The REMS program has some important benefits. The idea is to reduce the risk of heart failure, especially if it requires hospitalization.  For instance, if a patient's left ventricular ejection fraction(LVEF) drops below 50%, REMS mandates that the dosage of Camzyos be interrupted.

REMS data

REMS produces a lot of data, and that can help in assessing Camzyos. A group of scientists led by Matthew Martinez (Morristown Medical Center) and Milind Desai (Cleveland Clinic)  recently analyzed the data from nearly 6300 patients who received at least one dose of Camzyos (Martinez et al. 2025). There were 5573 patients with at least one report on their status (including the data from their echocardiogram).

Reduced obstruction

Camzyos provided significant reduction of obstruction. Initially all patients were obstructed (that is, they had gradients of at least 30mmHg after valsalva maneuvers). After 3 months on Camzyos, about 57% were no longer obstructed. After 6 months, about 70% had no obstruction.

Heart failure found early

But, how often did patients have problems indicating heart failure? LVEF at 50% or lower was recorded in 256 patients, 4.6% of the total. Symptomatic heart failure that required hospitalization occurred 71 (1.3%) times, and both occurred in 17 (0.3%) of the patients. When the analysis was restricted to patients who had been on Camzyos for at least a year, the numbers in these categories were roughly similar.

Conclusions

We can conclude several things. (1) Camzyos eliminates (or greatly reduces) obstruction in a majority, but not all, patients; (2) the risk of heart failure is relatively low; and (3) the REMS program is effective at protecting patients from severe heart failure and its consequences.

Literature cited

Martinez, M. W., D. Seto, M. Cheung, M. Coiro, N. Patel, A. Bastien, J. Lockman, S. Afsari, and M. Y. Desai. 2025. Mavacamten: Initial Insights From the Risk Evaluation and Mitigation Strategy Program.JACC: Advances4:101430. 

HCMA Blog

Lisa's Letter - July Newsletter

By Lisa Salberg • July 3, 2025
Summer, greetings to all our big-hearted friends As July approaches, our focus is already on the fall and preparing for some major events, including our annual meeting coming up in October. July will also find us on the west coast in Seattle conducting a regional patient education meeting as part of our big-hearted warrior tour. We have been following the generic drug quality issue in the United States very closely and encourage you to watch the series starting with our Hill briefing in April and following with the additional webinars with our partners at Medshadow and the People's Pharmacy, and of course the ProPublica series of articles. See them all here . We are happy to have a new team member on board - we welcome Pam as our coordinator of both our All Hearts Collaborative and Hearts and Minds project. Over the next few months, you're going to be learning more about these two amazing initiatives and how we are working to provide better services for big hearts regardless of where you live, so we are meeting all of our big-hearted friends where they are. Please stay tuned for updates from these projects coming soon. We are also creating new volunteer opportunities and engagements that we hope will make it easier for you to participate in spreading the message of the importance of diagnosis, the importance of community readiness related to CPR and AED use and, of course, helping patients get to their ultimate diagnosis and getting them on the proper treatment pathways. This July I would like to recognize all of the special birthdays in my family, including HCMA Center of Excellence coordinator, Stacey Titus-brown and my daughter Rebecca Salberg. It's a milestone birthday for Becca - it’s hard to believe I have a 30-year-old child. Wishing you all a happy and healthy summer. Go build some memories!
An official seal in black & red with white letters that say HCMS Recognized Centers of Excellence

July's Topic is COE Care at the HCMA

By Sabrina Cuddy • July 3, 2025
Getting HCM care at a HCMA Recognized Center of Excellence has a positive impact on patient's outcomes. Find out why in the post by Health Educator Sabrina Cuddy

Pro Publica Releases Results of FDA Investigation

By Erica Friedman • June 26, 2025
Investigative journalists Debbie Cenziper, Megan Rose, Brandon Roberts and Irena Hwang from Pro Publica and NPR have concluded a 14-month long investigation into the quality of generic drugs coming into the United States from overseas. Among the many voices that spoke up for American patients was HCMA Founder and CEO, Lisa Salberg who has felt the effects of low-quality drugs personally. Salberg believes that fixing this problem is something we can do, even in this time, when even health care is highly politicized. Read the key takeaways from ProPublica’s 14-month investigation into the FDA’s oversight of foreign drugmakers in Threat in Your Medicine Cabinet: The FDA’s Gamble on America’s Drugs .
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