Northern Ireland reviewing new heart drug after approval in England for thousands of NHS patients

A new once-daily tablet that could improve the lives of people living with a serious inherited heart condition is being considered for use in Northern Ireland after receiving approval for NHS patients in England.

Aficamten, marketed as MYQORZO, has been authorised by the Medicines and Healthcare products Regulatory Agency (MHRA) and simultaneously recommended by the National Institute for Health and Care Excellence (NICE) for adults with symptomatic obstructive hypertrophic cardiomyopathy (HCM).

The treatment is expected to become available to eligible patients in NHS England within 30 days, benefiting around 6,600 people.

However, while the medicine has not yet been approved for routine use in Northern Ireland, the Department of Health has confirmed it is now reviewing the NICE guidance under its normal assessment process.

What is hypertrophic cardiomyopathy?

Hypertrophic cardiomyopathy (HCM) is a condition in which the heart muscle becomes abnormally thick and stiff, making it harder for the heart to pump blood effectively.

It can cause symptoms including:

• breathlessness

• fatigue

• dizziness

• chest pain

• a racing or irregular heartbeat

Around one in every 500 people in the UK is thought to have HCM, with about half of cases inherited.

The new treatment is intended for adults whose symptoms are classed as New York Heart Association (NYHA) Class 2 or 3, meaning the condition limits everyday physical activity but does not usually cause symptoms while resting.

How the treatment works

Aficamten belongs to a newer class of medicines known as cardiac myosin inhibitors.

It works by reducing excessive contraction of the heart muscle, helping the heart pump more efficiently and reducing obstruction to blood flow.

Clinical trials found the medicine improves exercise capacity and reduces symptoms in people with obstructive HCM.

NICE also concluded it is likely to be as effective as mavacamten, the only other treatment of its kind currently available through the NHS, while potentially requiring fewer echocardiogram scans during dose adjustment.

Patients taking aficamten will still require regular heart monitoring throughout treatment.

Northern Ireland considering adoption

In a statement to Love Ballymena, Northern Ireland’s Department of Health stressed that NICE recommendations are routinely reviewed before being adopted locally.

The Department said treatments recommended by NICE for routine NHS use in England are generally considered for implementation within Northern Ireland’s Health and Social Care system.

It added that NICE published its final guidance recommending aficamten on 30 July 2026, and that the Department is now considering the appraisal through its established process.

That means the medicine is not yet routinely available in Northern Ireland, but it could become available if the guidance is formally endorsed following review.

Earlier access through closer working

The MHRA and NICE said closer collaboration between the two organisations allowed the medicine to be authorised and recommended at the same time, shortening the usual process.

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 licensing and value assessment decisions.”

Julian Beach, Executive Director of Healthcare Quality and Access at the MHRA, added:

“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.”

Patients welcome greater choice

Cardiomyopathy UK welcomed the decision, saying it gives patients another treatment option for a condition that can significantly affect everyday life.

Chief Executive Katharine McIntosh said the charity had shared evidence from patients during the NICE appraisal.

“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.”

She added that the outcome offers “greater choice for patients” and could allow more people living with obstructive HCM to benefit from treatment.

What patients should know

Aficamten is available as 5mg, 10mg, 15mg and 20mg tablets.

The medicine is approved as an addition to standard treatments such as beta blockers, certain calcium-channel blockers or disopyramide, or on its own if those medicines cannot be used.

Anyone taking the medicine will undergo regular echocardiograms to monitor heart function.

Anyone who experiences a suspected side effect should speak to their doctor, pharmacist or nurse and report it through the MHRA Yellow Card Scheme at https://yellowcard.mhra.gov.uk or via the MHRA Yellow Card app.

For more information about Northern Ireland’s process for adopting NICE Technology Appraisals, visit https://www.health-ni.gov.uk/sites/default/files/publications/health/doh-hsc-sqsd-12-22.pdf.

For now, patients in Northern Ireland will need to wait for the Department of Health to complete its review before any decision is made on making aficamten routinely available through Health and Social Care services.