A pill to treat obstructive hypertrophic cardiomyopathy (HCM) will be available to around 6,600 people in England following the simultaneous approval by the MHRA and NICE.
Around 1 in 500 people in the UK has 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.
Roughly half of the HCM cases is inherited. Clinical trials show that this once-daily pill, also known as MYQORZO, 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.
How it works
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.
Aficamten is available as film-coated tablets in strengths of 5 mg, 10 mg, 15 mg and 20 mg.
Patients receiving treatment will undergo regular monitoring of heart function, including echocardiograms, to assess how well their heart is working and to support safe use of the medicine.
Quicker approval
NICE has published final guidance two weeks faster than its standard processes due to efficiencies gained through a closer working relationship with the MHRA.
Helen Knight, director of medicines evaluation at NICE, said: "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.”
Julian Beach, MHRA Executive Director of Healthcare Quality and Access, said: "Today's authorisation of aficamten, alongside NICE's publication of final guidance, marks an important milestone for patients with obstructive hypertrophic cardiomyopathy and for the UK's medicines regulatory system.
"This demonstrates how closer working can help patients access innovative treatments sooner, while maintaining the robust standards of safety, quality and effectiveness that patients rightly expect.
Alternate treatment option
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.”
