Cytokinetics Announces FDA Acceptance of Aficamten for Obstructive Hypertrophic Cardiomyopathy
Tuesday, December 03, 2024
Cytokinetics has confirmed that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for aficamten.
Hypertrophic cardiomyopathy (HCM) is characterised by thickened heart muscle that impairs its ability to pump blood effectively, leading to reduced exercise capacity and other symptoms. It is the most common inherited cardiovascular condition, affecting hundreds of thousands in the United States, many of whom remain undiagnosed. The condition increases the risk of complications such as arrhythmias, heart failure, and sudden cardiac death, particularly in younger individuals and athletes.
This cardiac myosin inhibitor is intended to treat obstructive hypertrophic cardiomyopathy (HCM). The FDA has set a target decision date under the Prescription Drug User Fee Act (PDUFA) for 26 September 2025 and does not plan to convene an advisory committee meeting for this application.
The application is supported by data from the SEQUOIA-HCM Phase 3 trial, which showed aficamten improves exercise capacity, reduces symptoms, and enhances cardiac biomarkers in patients with obstructive HCM. The treatment demonstrated consistent benefits across all evaluated patient subgroups and was well-tolerated.
Key findings from the trial include a significant improvement in exercise capacity, measured by a 1.8 mL/kg/min increase in peak oxygen uptake over 24 weeks compared to no improvement in the placebo group. Secondary measures such as cardiac function, symptom scores, and treatment eligibility for septal reduction therapy also showed statistically significant benefits. Adverse events were lower among patients receiving aficamten compared to placebo, with no reports of heart failure exacerbation due to the treatment.
Aficamten works by targeting cardiac myosin to reduce myocardial hypercontractility, a key issue in hypertrophic cardiomyopathy. It has shown positive effects on heart structure and function without compromising systolic performance.
The FDA previously granted aficamten Orphan Drug Designation for symptomatic HCM in January 2021 and Breakthrough Therapy Designation for obstructive HCM in December 2021. The drug is also being reviewed by the National Medical Products Administration (NMPA) in China.
In addition to SEQUOIA-HCM, ongoing trials such as MAPLE-HCM, ACACIA-HCM, CEDAR-HCM, and FOREST-HCM are further investigating aficamten’s potential as a standalone treatment, its use in non-obstructive HCM, and its safety in paediatric populations.
Aficamten could represent a significant advancement in the management of obstructive HCM, offering new hope to patients affected by this challenging condition.
Source: cytokinetics.com
