The Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation for aficamten (MYQORZO, Cytokinetics, Inc.) for adults with symptomatic obstructive hypertrophic cardiomyopathy (HCM), alongside publication of final guidance from the National Institute for Health and Care Excellence (NICE). 

The coordinated decisions mean around 6600 eligible people in England could now gain access to the once-daily treatment. Aficamten is the second cardiac myosin inhibitor available in the UK, alongside mavacamten. 

Obstructive HCM affects about 1 in 500 people in the UK. The condition causes abnormal thickening and stiffening of the heart muscle, which can obstruct blood flow and lead to symptoms including breathlessness, fatigue, chest pain, dizziness, and palpitations that substantially affect quality of life.

New Option for Symptomatic Obstructive HCM 

Aficamten is a targeted, reversible cardiac myosin inhibitor. By binding directly to the myosin protein within myocardial cells, it decreases hypercontractility, alleviates left ventricular outflow tract obstruction, and improves overall cardiac efficiency.

The drug is indicated for adults with symptomatic obstructive HCM classified as New York Heart Association (NYHA) class II or III, a scale used by clinicians to grade how much a cardiac condition limits daily activity. It is recommended as an add-on to standard therapy, including beta-blockers, certain calcium-channel blockers, or disopyramide, or as monotherapy where these cannot be used.

Katharine McIntosh, CEO of Cardiomyopathy UK, welcomed the decision, saying an additional option was important because mavacamten is currently the only treatment of its kind available. She added that “because aficamten may need fewer echocardiograms in the early stages of treatment, it could represent a more accessible treatment route.” 

Improved Exercise Capacity 

The authorisation was based on the phase 3 SEQUOIA-HCM trial, in which 282 patients (mean age, 59.1 years; 59.2% men) with symptomatic obstructive HCM were randomly assigned to receive aficamten (starting dose 5 mg, titrated up to 20 mg) or placebo for 24 weeks.

By week 24, aficamten significantly improved exercise capacity, increasing peak oxygen uptake by a mean of 1.8 mL/kg/min, compared with no meaningful change in the placebo group (least-squares mean between-group difference, 1.7 mL/kg/min; P < .001). The treatment also met all 10 prespecified secondary endpoints, with significant improvements in health status as measured by the Kansas City Cardiomyopathy Questionnaire clinical summary score, NYHA functional class, and other disease-related outcomes.

The incidence of adverse events was comparable between the aficamten and placebo groups.

Dosing and Monitoring 

Aficamten is supplied as film-coated tablets in 5 mg, 10 mg, 15 mg, and 20 mg strengths. Patients will require regular cardiac monitoring, including echocardiograms, to assess heart function and guide safe dosing. 

NICE said aficamten offers a more flexible echocardiography schedule and a shorter titration period than mavacamten, which could be beneficial for patients and for services managing monitoring capacity.

Cost, NHS Rollout, and Aligned MHRA-NICE Decisions 

Aficamten has a list price of £1180.52 for a 28-tablet pack, excluding VAT. The company has established a commercial arrangement that makes the therapy available to the NHS with a discount, though the size of the discount remains commercially confidential. 

The simultaneous MHRA authorisation and NICE final guidance reflect closer collaboration between the two organisations to accelerate patient access to new medicines. 

NICE assessed aficamten through a cost-comparison process against mavacamten, which is already recommended for the same population. In its guidance, NICE said indirect comparisons suggest the two treatments are likely to be similarly effective and that aficamten’s costs are similar to or lower than those of the existing treatment. NICE said closer working with the MHRA meant this guidance was published 2 weeks faster than under its standard processes. It added that guidance for medicines overall has been published 30% faster since April 2024, as part of its wider transformation programme.

Helen Knight, director of medicines evaluation at NICE, said the coordinated decision showed the value of closer working between NICE and the MHRA, helping safe and effective medicines reach patients earlier by aligning licensing and value assessment decisions.

Julian Beach, MHRA executive director of healthcare quality and access, said, “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,” he added.

NHS England will make aficamten available within 30 days of NICE publishing its final guidance.

As with all newly licensed therapies, aficamten remains under close postmarketing safety monitoring by the MHRA. Healthcare professionals are advised to review the full Summary of Product Characteristics on the MHRA website and report any suspected adverse reactions via the Yellow Card scheme.