Milind Desai, MD: Real-World Safety Data Supports Mavacamten for oHCM

Real-world results from the Risk Evaluation and Mitigation Strategy (REMS) program confirmed mavacamten, a cardiac myosin inhibitor for obstructive hypertrophic cardiomyopathy (oHCM), maintained a favorable safety profile in more than 6,000 real-world patients.

Presented at the American Heart Association (AHA) Scientific Sessions 2024, data collected between April 2022 and February 2024 revealed that temporary treatment interruption due to reduced left ventricular ejection fraction (LVEF <50%) was rare, occurring in only 4.6% of patients, while HF requiring hospitalization (HFH) was even less frequent (1.3%).

“Overall, all the REMS objectives with regards to the mavacamten program were achieved based on the current report,” presenting investigator Milind Y. Desai, MD, MBA, a professor of medicine at the Cleveland Clinic Lerner College of Medicine, told HCPLive. “Of course, REMS is not a clinical trial, we don’t have extensive data, but that is not what this program was designed [to do]. Every patient in the United States is represented here."

Among the 1,929 patients treated with mavacamten for at least 1 year, 4.04% experienced LVEF reductions to <50% and 0.21% had concurrent HFH, often linked to confounders like atrial fibrillation. Across 29,111 health status forms submitted during the study, LVEF <50% was reported in 0.9% of cases, and HFH occurred in only 0.3%. Additionally, drug interaction monitoring—part of the REMS protocol—led to dose adjustments in 0.25% of cases and discontinuation of interacting medications in 0.32%.

Overall, these findings reinforce mavacamten’s safety and tolerability for patients with symptomatic NYHA class II-III oHCM, even with prolonged use. Clinicians can be reassured by these real-world outcomes, demonstrating effective risk management under the REMS program. Further dosing analyses are expected to provide additional insights into optimizing long-term treatment strategies.

“The next programs will inevitably require a REMS program and they have a playbook they can adapt off of the mavacamten experience,” Desai told HCPLive. “I would imagine the future set of cardiologists or practicing clinicians who may have to deal with the REMS program, we have a huge contemporary lesson to learn from."

Disclosures: Desai reports relevant disclosures with Bristol Myers Squibb, Cytokinetics, Edgewise, and others.

Reference

_{Desai MY, Seto D, Cheung M, Coiro M, et al. Mavacamten: Real-World Experience from 22 Months of the Risk Evaluation and Mitigation Strategy (REMS) Program. Presented at the American Heart Association (AHA) Scientific Sessions 2024. Chicago, Illinois. November 16-18, 2024.}