International Study Shows Mavacamten Monotherapy Improves Heart Function in HCM Patients

Researchers find single targeted therapy can offer strong clinical benefits while simplifying care for common inherited heart disease.

Published on Feb. 17, 2026

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Researchers from the University of Alabama at Birmingham Marnix E. Heersink School of Medicine and collaborators around the world have published a study showing that mavacamten, used as a standalone therapy without beta-blockers or calcium-channel blockers, significantly improves symptoms and cardiac obstruction in people with obstructive hypertrophic cardiomyopathy (HCM), a leading cause of heart-related disability in younger adults.

Why it matters

HCM is a hereditary condition where the heart muscle abnormally thickens and stiffens, blocking blood flow. Until now, treatment has relied on long-standing drugs like beta-blockers, which help symptoms but do not directly target the biology of the disease. This study provides the first global evidence that mavacamten alone can meaningfully reduce outflow tract obstruction and improve symptoms in routine clinical practice, offering a new targeted therapy option.

The details

The present study draws on real‑world clinical data from 278 patients across five countries in the mavaCamten ObservationaL evIdence Global cOnsortium (COLLIGO-HCM). Across the nine-month follow-up period, 60 percent of patients treated with mavacamten monotherapy improved by at least one New York Heart Association class, and outflow tract gradients fell by 35-59 mmHg on average, bringing most patients below guideline thresholds for obstruction. Importantly, systolic function remained stable, and treatment interruptions for reduced ejection fraction were uncommon and largely reversible.

  • The study was published on February 16, 2026 in Circulation: Genomic and Precision Medicine.

The players

Pankaj Arora

Senior author and director of the UAB Cardiogenomics Clinic.

Garima Arora

Co-author and co-director of the UAB Cardiogenomics Clinic.

University of Alabama at Birmingham Marnix E. Heersink School of Medicine

The institution where the lead researchers are based.

mavaCamten ObservationaL evIdence Global cOnsortium (COLLIGO-HCM)

The global consortium that provided the real-world clinical data for the study.

Circulation: Genomic and Precision Medicine

The medical journal where the study was published.

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What they’re saying

“The study provides the first global evidence that mavacamten alone can meaningfully reduce outflow tract obstruction and improve symptoms in routine clinical practice.”

— Pankaj Arora, Director of the UAB Cardiogenomics Clinic

“These findings show that many patients may not need multiple medications. A single targeted therapy can offer strong clinical benefits while simplifying care.”

— Garima Arora, Co-director of the UAB Cardiogenomics Clinic

“This is more than a therapeutic advance; it is a glimpse into the future of precision cardiology. Real-world data like these help clinicians make confident, patient-centered decisions.”

— Pankaj Arora, Director of the UAB Cardiogenomics Clinic

What’s next

According to the authors, continued expansion of access to genetic testing, advanced imaging and precision therapeutics could ensure that patients across all communities benefit from the growing era of targeted cardiovascular care.

The takeaway

This study provides promising evidence that a single targeted therapy, mavacamten, can significantly improve heart function and symptoms for patients with the common inherited heart condition hypertrophic cardiomyopathy, potentially offering a new treatment option that simplifies care.