Around 6,600 people in England with a serious heart condition will be able to access a new treatment following simultaneous MHRA authorisation and NICE approval.

Aficamten, also known as MYQORZO, is a once-daily pill for people with obstructive hypertrophic cardiomyopathy (HCM) – a condition where the heart muscle becomes thickened and stiff, making it harder for the heart to pump blood around the body. It can cause exhaustion, dizziness, breathlessness, chest pain and a racing or irregular heartbeat. Around 1 in 500 people in the UK has HCM, and in roughly half of cases it is inherited.

Clinical trials show aficamten helps people exercise more easily and reduces their symptoms. It is likely to be as effective as mavacamten, the other NICE-approved treatment for HCM, and patients may need fewer monitoring scans to find the right dose.

The active substance, aficamten, is a reversible cardiac myosin inhibitor which binds to the protein myosin in heart muscle cells, reducing the excessive contraction of the heart muscle. This helps the heart pump more smoothly, reduces obstruction to blood flow and may improve symptoms of obstructive hypertrophic cardiomyopathy.

Earlier access to new medicines

NICE used a cost comparison approach for this drug, where to get a positive recommendation, the company needed to demonstrate the costs for aficamten are similar to or lower than those for mavacamten for treating symptomatic HCM, which is recommended by NICE for this population and is already in use in the NHS.
NICE has published final guidance two weeks faster than its standard processes due to efficiencies gained through a closer working relationship with the MHRA.
NHS England will make the treatment available within 30 days of NICE publishing its final guidance. A confidential discount has been agreed with the company, to make aficamten available to the NHS at a reduced cost.

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.”