NICE recommends heart drug aficamten with simultaneous MHRA approval

A new treatment for obstructive hypertrophic cardiomyopathy (HCM) has been simultaneously recommended for use on the NHS in England and Wales by the National Institute for Health and Care Excellence (NICE) and approved by the Medicines and Healthcare products Regulatory Agency (MHRA).

In final guidance, published on 30 July 2026, NICE recommended aficamten (Myqorzo; Cytokinetics UK) to treat adults whose HCM — a condition in which the heart muscle thickens — causes slight-to-marked limitations on physical activity.

Aficamten, which is available in tablet form, reversibly binds to myosin in heart muscle cells and reduces excessive contraction of the heart muscle, the guidance said. It added that a starting dose of aficamten 5mg daily is recommended for most people, which is to be increased by 5mg every 2 to 8 weeks to a maintenance dose, or a maximum dose of 20mg.

According to NICE, HCM affects around 1 in 500 people in the UK and around half of cases are inherited.

The British Heart Foundation has reported that some patients are asymptomatic, but others experience symptoms, including chest pain, lightheadedness or fainting, palpitations, shortness of breath, fatigue and swelling of the legs, feet, ankles and abdomen.

In a statement published alongside the final guidance, NICE said: “[Aficamten] 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.”

In addition, aficamten has a shorter titration period compared with mavacamten, it continued.

Under the final guidance, the drug can be used for adults with HCM and a New York Heart Association class of two to three, as an add-on treatment alongside other medicines including beta-blockers, non-dihydropyridine calcium-channel blockers or disopyramide or used alone if the patient cannot take other treatments.

Katharine McIntosh, chief executive of the charity Cardiomyopathy UK, commented: “This outcome means greater choice for patients and the opportunity for more people living with HCM to benefit from treatment, feel better and live well.”

Paul Wright, consultant cardiovascular pharmacist at Barts Heart Centre, Barts Health NHS Trust, said the drug’s approval “was really exciting news and occurred quicker than expected”, thanks to the collaborative approach from the MHRA and NICE.

“The introduction of the second in class (cardiac myosin inhibitor) will be warmly welcomed by physicians and patients alike with benefits of relaxed echo requirements (wider intervals between dose titrations and initiation) and no requirement for genetic phenotyping prior to starting therapy unlike mavacamten, the current agent,” he added.

“Currently, these agents are licensed for treating obstructive hypertrophic cardiomyopathy (oHCM) — we are waiting for results to be shared in trials in non-obstructive HCM that may demonstrate benefits in this cohort and thus further improving outcomes in individuals with HCM.”

In its statement, NICE also said that the regulator had produced its final guidance for the drug two weeks faster than usual, owing to a new process that sees it working more closely with the MHRA.

The ten-year health plan for England, published in 2025, set out plans for NICE and the MHRA to work together on a “new joint process, supported by information sharing and joint scientific advice, that will boost the speed of decisions and cut administrative burdens for the system and industry”.

Julian Beach, executive director of healthcare quality and access at the MHRA, said: “[The joint approval and guidance] 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.”