FDA Updates Mavacamten Label in Obstructive Hypertrophic Cardiomyopathy

The US Food and Drug Administration (FDA) has updated the label for mavacamten (CAMZYOS) 2.5 mg, 5 mg, 10 mg, and 15 mg capsules for the treatment of adults with symptomatic New York Heart Association (NYHA) Class II-III obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms.1

Announced by Bristol Myers Squibb on April 17, 2025, the prescribing information has been updated to simplify the required echo monitoring for eligible patients in the maintenance phase and expand patient eligibility by reducing contraindications.

“With robust clinical and real-world data and more than 15,000 patients prescribed [mavacamten] in the US, this medicine has redefined the treatment landscape for symptomatic obstructive HCM and can have a significant impact for patients living with the condition,” said Al Reba, senior vice president of cardiovascular & immunology commercialization at Bristol Myers Squibb. “Simplying treatment by reducing the frequency of echo monitoring not only improves the patient experience, but will also save time for cardiologists, allowing them to treat more patients.”1

With initial regulatory approval in April 2022, mavacamten marked the first and only cardiac myosin inhibitor approved to improve functional capacity and symptoms in NYHA class II-III oHCM. Results from the EXPLORER-HCM trial showed mavacamten improved exercise capacity, left ventricular outflow tract (LVOT) obstruction, NYHA functional class, and health status.2

In June 2023, Bristol Myers Squibb announced an approved update to mavacamten’s label based on positive data from VALOR-HCM. The update reflected the agent's ability to reduce the need or eligibility for septal reduction therapy (SRT) in patients with obstructive HCM.3

With this current update, the FDA has reduced the frequency of required echo monitoring from once every 12 weeks to every 6 months for individuals with left ventricular ejection fraction (LVEF) ≥55% and a Valsalva LVOT gradience <30 mmHg (or Valsalva LVOT ≥30 mmHg without up-titration) who reached the maintenance phase at 12 weeks or later.1

Further updates eliminated the contraindication of mavacamten with moderate CYP2C19 inhibitors and strong CYP3A4 inhibitors, adjusted due to drug interaction. Bristol Myers Squibb indicated this change would offer physicians more flexibility in prescribing mavacamten to a broader group of eligible patients, with revised dosing and monitoring.1

According to the release, this approved label update was supported by long-term clinical and real-world data, including the mavacamten Risk Evaluation and Mitigation Strategy (REMS) program, real-world data in three single-center studies, and current clinical data reporting the safety profile of mavacamten across 3.5 years.1

Bristol Myers Squibb also noted mavacamten has been included in European Society of Cardiology (ESC) and American Heart Association (AHA)/American College of Cardiology (ACC)/Multisociety clinical guidelines as a recommended option for patients with persistent symptoms on a first-line therapy.1

“In addition to the established efficacy of [mavacamten], these meaningful updates to the label reinforce the strong safety profile of the therapy,” Reba added.1

On April 14, 2025, the company announced mavacamten missed dual primary endpoints in the Phase 3 ODYSSEY-HCM trial, which assessed the cardiac myosin inhibitor against placebo in adults with symptomatic NYHA class II-III non-obstructive HCM. Mavacamten did not achieve statistical significance in changes from baseline to Week 48 in the Kansas City Cardiomyopathy Questionnaire (KCCQ) Clinical Summary Score and peak oxygen consumption, but reported no new safety signals.4

References

1. U.S. Food and Drug Administration Updates CAMZYOS® (mavacamten) Label to Reduce Echocardiography Monitoring Requirements and Contraindications. Bristol Myers Squibb. April 17, 2025. Accessed April 18, 2025. https://news.bms.com/news/corporate-financial/2025/U-S--Food-and-Drug-Administration-Updates-CAMZYOS-mavacamten-Label-to-Reduce-Echocardiography-Monitoring-Requirements-and-Contraindications/default.aspx.

2. Campbell P. FDA approves Mavacamten (Camzyos) for obstructive hypertrophic cardiomyopathy. HCP Live. November 7, 2023. Accessed April 18, 2025. https://www.hcplive.com/view/fda-approves-mavacamten-for-obstructive-hypertrophic-cardiomyopathy.

3. Campbell P. FDA approves updated label for Mavacamten in obstructive hypertrophic cardiomyopathy. HCP Live. June 16, 2023. Accessed April 18, 2025. https://www.hcplive.com/view/fda-approves-updated-label-for-mavacamten-in-obstructive-hypertrophic-cardiomyopathy.

4. Iapoce C. Mavacamten falls short in phase 3 trial for non-obstructive HCM. HCP Live. April 15, 2025. Accessed April 18, 2025. https://www.hcplive.com/view/mavacamten-falls-short-in-phase-3-trial-for-non-obstructive-hcm