Thousands of patients to benefit from new daily pill for serious heart condition following co-ordinated MHRA and NICE decisions
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 today.
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.
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.
NICE is publishing guidance 30% faster for medicines and 9% faster for healthtech than before April 2024, following changes made through our transformation programme.
Katharine McIntosh, CEO of Cardiomyopathy UK, said: “We’re delighted that aficamten is going to be made available to people living with obstructive hypertrophic cardiomyopathy (HCM). As part of the NICE appraisal, we submitted insights from our patient community, including the impact of symptoms, experiences of NHS treatment and care, and what another treatment option could mean.
“Obstructive HCM can have a considerable impact, with symptoms such as breathlessness, fatigue and a reduced ability to exercise making day-to-day life significantly more challenging. Mavacamten is currently the only treatment of this kind available, so having an additional option is very welcome. And because aficamten may need fewer echocardiograms in the early stages of treatment, it could represent a more accessible treatment route.
“This outcome means greater choice for patients and the opportunity for more people living with obstructive HCM to benefit from treatment, feel better and live well.”
About the treatment
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.
Excerpt from the official page. The original is authoritative. Continue reading at UK NICE ↗