Around 6,600 people in England living with a debilitating heart condition are set to benefit from a new treatment after receiving simultaneous approval from the Medicines and Healthcare products Regulatory Agency and the National Institute for Health and Care Excellence.
Aficamten, marketed as MYQORZO, has been authorised for use in adults with symptomatic obstructive hypertrophic cardiomyopathy (HCM), a condition that causes the heart muscle to become abnormally thick and stiff. The disease can significantly restrict blood flow and lead to symptoms including breathlessness, fatigue, dizziness, chest pain and irregular heart rhythms.
Hypertrophic cardiomyopathy affects around one in 500 people in the UK, with approximately half of all cases linked to inherited genetic factors.
The newly approved once-daily tablet offers patients another treatment option alongside mavacamten, which is already available through the NHS. Clinical trial evidence suggests aficamten improves exercise capacity and reduces symptoms, helping patients manage the impact of the condition more effectively.
Importantly, aficamten is expected to provide comparable benefits to mavacamten while potentially requiring fewer monitoring scans to identify the most appropriate dose, offering a more streamlined treatment experience for some patients.
NICE approved the medicine through its cost-comparison process. Under this approach, the manufacturer was required to demonstrate that the overall cost of treatment was comparable to, or lower than, the cost of mavacamten, the existing NICE-recommended therapy for this patient group.
The decision also highlights growing collaboration between NICE and the MHRA. Through closer alignment of licensing and health technology assessment processes, final NICE guidance was issued two weeks faster than standard timelines, helping patients gain access to innovative medicines more quickly.
The latest approval reflects wider improvements across NICE's medicines evaluation programme. Since changes introduced through its transformation programme in April 2024, NICE reports that it is now publishing guidance 30% faster for medicines and 9% faster for health technologies.
Helen Knight, Director of Medicines Evaluation at NICE, said:
“We’re pleased to be able to recommend a treatment that has the potential to alter the course of obstructive hypertrophic cardiomyopathy and offers new hope to people with this serious condition.
“This treatment being approved demonstrates the value of closer working between NICE and the MHRA, ensuring that safe, effective medicines get to people earlier by aligning licencing and value assessment decisions.”
Aficamten works by acting as a reversible cardiac myosin inhibitor. It binds to the myosin protein within heart muscle cells, reducing excessive contraction of the heart muscle. This action improves blood flow through the heart, eases obstruction and may help reduce symptoms associated with obstructive HCM.
The medicine will be available as film-coated tablets in strengths of 5mg, 10mg, 15mg and 20mg. Patients prescribed aficamten will continue to undergo regular heart function monitoring, including echocardiograms, to ensure treatment remains safe and effective.
The approval marks another step forward in the treatment of hypertrophic cardiomyopathy and demonstrates how closer cooperation between regulators and health technology assessors can accelerate patient access to innovative therapies across the NHS.
Image credit: iStock
