Mavacamten has been approved in the European Union since 2023 for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (HOCM). In this condition, the heart muscle is thickened and can obstruct blood flow out of the left ventricle. Mavacamten targets a mechanism underlying the disease, reducing excessive contraction of the heart muscle.
The efficacy and safety of the drug were investigated in controlled clinical trials prior to approval. However, such trials are conducted under tightly defined conditions and in selected patient populations. Registries such as TORCH-Plus make it possible to subsequently examine the outcomes observed with a therapy under the conditions of routine patient care.
Data from 15 German cardiomyopathy centres
For the current interim analysis, data from 332 patients with symptomatic HOCM who had started treatment with mavacamten were evaluated. They were treated at 15 specialist cardiomyopathy centres across Germany.
After twelve months, 65 per cent of the patients studied had improved by at least one NYHA class. NYHA classes describe the degree to which physical capacity is limited in people with heart disease. In addition, obstruction of blood flow from the left ventricle decreased, and levels of the cardiac stress marker NT-proBNP fell. Symptoms and everyday limitations reported by patients themselves also decreased.
"TORCH Mava demonstrates the potential of collaboration between academic research and industry. With the TORCH-Plus infrastructure, we can jointly investigate how new therapies perform in routine clinical practice after approval. Data from specialist centres across Germany complement the findings from clinical trials with insights from real-world patient care," says Professor Benjamin Meder, lead scientist of TORCH-Plus-DZHK21 and Head of the Institute for Cardiomyopathies at Heidelberg University Hospital.
Research infrastructure for industry collaborations
TORCH-Plus-DZHK21 is led by the DZHK and now includes more than 4,700 patients with cardiomyopathies. The registry brings together clinical data, imaging findings, biomarkers and genetic information, and follows patients over extended periods. Through this research infrastructure, the DZHK is also creating the basis for collaborations with industry that generate additional insights into new therapies in routine care. As part of the collaboration on mavacamten, Bristol Myers Squibb is funding the analyses and other costs arising from the study.
More information on the registry: TranslatiOnal Registry for CardiomyopatHies-Plus (TORCH-Plus-DZHK21)
Scientific contact: Dr med. Jan Kölemen, Department of Cardiology, Angiology and Pneumology, Heidelberg University Hospital, Jan.Koelemen[at]med.uni-heidelberg.de
