A new drug, aficamten, significantly improved exercise capacity and reduced symptoms in patients with obstructive hypertrophic cardiomyopathy, a common genetic heart condition, according to a recent study published in the New England Journal of Medicine.

Hypertrophic cardiomyopathy (HCM) is characterized by a thickened heart muscle that can obstruct blood flow, leading to shortness of breath, chest pain, and reduced ability to exercise. Current treatments, such as surgery and medication, have limitations and side effects.

The phase 3 clinical trial, SEQUOIA-HCM, included 282 patients with symptomatic obstructive HCM. Mean age was 59 and almost 60% were male.

Participants were randomly assigned to receive either aficamten or a placebo for 24 weeks. The results showed that patients treated with aficamten experienced a significant improvement in peak oxygen uptake (1.7 ml per kilogram per minute) during exercise, a key measure of cardiovascular fitness. In the placebo group, an update of 0.0 ml per kilogram per minute was found.

In addition to improving exercise capacity, aficamten showed better symptom control, with more patients reporting a reduction in their New York Heart Association functional class, which measures the severity of heart failure symptoms. The drug also significantly reduced the pressure gradient in the heart, a hallmark of obstructive HCM.

The safety profile of aficamten appeared to be favorable, with no significant differences in adverse events between the treatment and placebo groups. However, longer-term studies will be needed to fully assess the drug's safety and efficacy.

If approved by regulatory authorities, aficamten could offer a new treatment option for patients with obstructive HCM, potentially improving their quality noted further research is needed to determine the optimal use of aficamten in clinical practice and to identify which patients are most likely to benefit from the therapy.

Several researchers reported receiving grants and personal fees from various organizations, including pharmaceutical companies.