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.

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.

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.”

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.

“We’re assured that the appropriate regulatory standards for the approval of this medicine have been met and, as with all medicines, we will keep its safety under close review.”

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 aficamten

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.

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.

As with any medicine, the MHRA will keep the safety and effectiveness of aficamten under close review. Anyone who suspects they are having a side effect from this medicine is encouraged to talk to their doctor, pharmacist or nurse and report it directly to the Yellow Card scheme, either through the website (https://yellowcard.mhra.gov.uk) or by searching the Google Play or Apple App stores for MHRA Yellow Card.

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.

Read the final guidance for aficamten for treating symptomatic obstructive hypertrophic cardiomyopathy on the NICE website.

Notes

• Aficamten, also known as MYQORZO, is made by Cytokinetics, Inc. It received marketing authorisation on Wednesday, 29 July.
• It is approved for adults with symptomatic obstructive HCM who have a New York Heart Association (NYHA) classification of 2 or 3. NYHA classification is a system used by doctors to describe how much a heart condition limits a person’s daily activities.
• It is recommended as an add-on to standard care - including beta-blockers, certain calcium-channel blockers, or disopyramide - or on its own if those treatments cannot be used.
• The Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for regulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe.  All our work is underpinned by robust and fact-based judgements to ensure that the benefits justify any risks.
• The MHRA is an executive agency of the Department of Health and Social Care.
• For media enquiries, please contact the newscentre@mhra.gov.uk, or call on 020 3080 7651 or contact the NICE Press Office on 0797 397 0534 or by emailing pressoffice@nice.org.uk.
• NICE’s core purpose is to help practitioners and commissioners get the best care to people, fast, while ensuring value for the taxpayer.
• We do this by producing useful and usable guidance for health and care practitioners, providing rigorous, independent assessment of complex evidence for new health technologies, developing recommendations that focus on what matters most and drive innovation into the hands of health and care practitioners, encouraging the uptake of best practice to improve outcomes for everyone.
• To find out more about what we do, visit our website: www.nice.org.uk and follow us on social media.

source: GOV