Cardiology Month in Review: April 2025

April marked a month of progress and recalibration in cardiology, as new trial data and US Food and Drug Administration (FDA) decisions underscored both the promise and complexity of advancing cardiovascular care.

From gene editing to device-based therapies, developments pointed toward more precise and durable solutions for lipid management and hypertension. Early-phase results for a genetic editing medicine and a label expansion for a structural heart disease drug reflected growing momentum behind personalized treatment approaches, even as setbacks reminded the field of the hurdles that remain.

As innovation accelerates, the interplay between clinical trial evidence, regulatory action, and real-world application continues to shape the future of cardiovascular medicine.

Here’s a recap of what happened in cardiology in April:

FDA Grants Fast Track Designation to VERVE-102 Gene Therapy

On April 11, 2025, the FDA awarded Fast Track designation to Verve Therapeutics’ VERVE-102 gene editing medicine to lower low-density lipoprotein cholesterol (LDL-C) levels in individuals with hyperlipidemia and high cardiovascular risk. The regulatory agency cleared the Investigational New Drug (IND) application for VERVE-102 in March 2025, when the company provided interim data from the dose–escalation part of the Phase 1b Heart-2 trial.

VERVE-102 Safely Cuts LDL-C Levels in Early Phase 1b Results

VERVE-102 later showed promising efficacy for the treatment of patients with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease (CAD) in the Heart-2 trial. A single infusion of the in vivo gene editing medicine achieved dose-dependent reductions in blood PCSK9 and LDL-C, with a mean LDL-C reduction of ≥50% and a maximum decrease of nearly 70% at the highest dose.

Mavacamten Falls Short in Phase 3 Trial for Non-Obstructive HCM

Results from the Phase 3 ODYSSEY-HCM trial found Bristol Myers Squibb’s mavacamten (Camzyos) missed dual primary endpoints against placebo for the treatment of adult patients with symptomatic New York Heart Association (NYHA) class II-III non-obstructive hypertrophic cardiomyopathy (HCM). The cardiac myosin inhibitor did not achieve statistical significance in changes from baseline to Week 48 in the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score and peak oxygen consumption, but reported no new safety signals.

Combination of Statins, Ezetimibe Could Prevent Thousands of Heart Attacks

A lipid-lowering therapy combination of statins and ezetimibe could prevent thousands of further cardiovascular events among patients hospitalized with myocardial infarction (MI). Patients who received combination therapy within 12 weeks were able to lower cholesterol to the target level early, had a better prognosis, and had less risk of new cardiovascular events and death.

FDA Updates Mavacamten Label in Obstructive Hypertrophic Cardiomyopathy

On April 17, 2025, the FDA updated the label for mavacamten (CAMZYOS) 2.5 mg, 5 mg, 10 mg, and 15 mg capsules for the treatment of adults with symptomatic NYHA Class II-III obstructive HCM to improve functional capacity and symptoms. The prescribing information simplified the required echo monitoring for eligible patients in the maintenance phase and expanded patient eligibility by reducing contraindications.

FDA Awards Breakthrough Device Designation to AVIM Therapy for Hypertension

On April 22, 2025, the FDA granted Breakthrough Device designation to Orchestra BioMed Holdings, Inc.’s implantable pacemaker system delivering atrioventricular interval modulation (AVIM) therapy to patients with elevated atherosclerotic cardiovascular disease (ASCVD) risk and uncontrolled hypertension. The AVIM therapy is labeled under an FDA investigational device exemption in the global BACKBEAT trial, conducted in collaboration with Medtronic.

FDA Delays Decision on Elamipretide for Barth Syndrome

On April 30, 2025, the FDA delayed the target action date for the New Drug Application (NDA) for the investigational medicine, elamipretide, for Barth syndrome. Labelling discussions were initiated with the regulatory agency, but a revised action date was not communicated to Stealth BioTherapeutics for the ultra-rare, life-threatening disease.