An international clinical trial found that individuals with obstructive hypertrophic cardiomyopathy showed significant improvement in their ability to use oxygen while exercising after taking the investigational drug aficamten, as reported in the New England Journal of Medicine and at the European Society of Cardiology’s Heart Failure 2024 meeting. Oregon Health & Science University participated in the Phase 3 trial, with 19 patients enrolled through the institution. The study showed promising results for aficamten as a potential treatment for HCM, a common heart condition that thickens heart muscles and reduces the heart’s ability to function properly, resulting in shortness of breath and decreased exercise capacity.
Hypertrophic cardiomyopathy affects about 1 in 500 people and is a leading cause of sudden death in young individuals and athletes. The obstructive form of HCM, which restricts blood flow out of the heart, can significantly impact a person’s quality of life. The study compared the effects of aficamten to a placebo in 282 adults, demonstrating that those who received the experimental drug showed a notable increase in their maximum oxygen uptake during exercise. This improvement can enhance physical activity and reduce the risk of heart failure, the need for a heart transplant, and mortality associated with reduced oxygen uptake in HCM patients.
Current non-drug treatment options for obstructive HCM include surgical procedures to remove excess heart muscle, while mavacamten was approved by the FDA in 2022 as the first drug specifically targeting the condition’s underlying cause. However, mavacamten comes with potential risks, such as increased heart failure and interactions with other medications, requiring intensive monitoring for patients. OHSU has been actively involved in researching and exploring new treatment options for HCM, including participating in aficamten trials for different forms of the condition and patient populations, including children.
The successful results of the aficamten trial represent a significant advancement in the treatment of HCM, offering patients more therapeutic options beyond traditional surgical interventions. Along with surgical and procedural therapies, patients now have access to FDA-approved medications like mavacamten and investigational drugs like aficamten through clinical trials. OHSU’s involvement in multiple research studies on HCM treatment underscores the institution’s commitment to advancing medical knowledge and improving patient outcomes in the field of cardiology.
Cardiologist Ahmad Masri, M.D., M.S., who co-authored the study, emphasized the importance of these new treatment options for patients with obstructive hypertrophic cardiomyopathy. With the ability to improve oxygen utilization during exercise, aficamten could enhance patients’ daily activities and quality of life. The ongoing research on aficamten and other potential therapies for HCM signals a promising future for individuals living with this challenging heart condition. As advancements in medical science continue to evolve, patients can benefit from a wider range of treatment options to manage their condition effectively and improve their overall cardiovascular health.