MHRA authorises and NICE recommends aficamten (MYQORZO) for adults with symptomatic obstructive hypertrophic cardiomyopathy, expanding NHS treatment access in England.
Written By: Anamika Koshti, PharmD
Reviewed By: Pharmacally Editorial Team
The Medicines and Healthcare products Regulatory Agency (MHRA) licensed aficamten, marketed as MYQORZO, on 29 July 2026, while the National Institute for Health and Care Excellence (NICE) published final guidance recommending the medicine on the same day. The coordinated decision enables around 6,600 eligible adults in England with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to receive the once-daily oral treatment through the NHS. Developed by Cytokinetics, Inc., aficamten becomes the second targeted cardiac myosin inhibitor available for this condition in England.
Under the MHRA licence, aficamten is indicated for adults with symptomatic obstructive hypertrophic cardiomyopathy (New York Heart Association [NYHA] Class II-III). NICE recommends the medicine for adults whose symptoms persist despite optimally tolerated standard therapy, providing an additional treatment option alongside mavacamten.
About Obstructive Hypertrophic Cardiomyopathy
Hypertrophic cardiomyopathy is a disease in which the heart muscle becomes abnormally thick, making it more difficult for the heart to pump blood effectively. In the obstructive form of the disease, the thickened heart muscle narrows the left ventricular outflow tract, partially blocking blood flow from the heart. Patients commonly experience shortness of breath, fatigue, chest pain, dizziness, palpitations and exercise intolerance, which can substantially affect quality of life.
Approximately 1 in 500 people in the UK is estimated to have hypertrophic cardiomyopathy, and around half of cases are inherited.
How Aficamten Works
Aficamten is a selective, reversible cardiac myosin inhibitor that reduces excessive actin-myosin cross-bridge formation responsible for myocardial hypercontractility. By decreasing excessive cardiac muscle contraction, the medicine reduces left ventricular outflow tract obstruction and improves the heart’s ability to pump blood more efficiently.
MYQORZO is available as film-coated tablets in 5 mg, 10 mg, 15 mg and 20 mg strengths. Treatment requires echocardiographic assessment before and during therapy to monitor left ventricular ejection fraction, guide dose adjustments and ensure continued safe use.
Clinical Evidence from the SEQUOIA-HCM Trial
The MHRA approval was supported by results from the pivotal Phase III SEQUOIA-HCM trial (NCT05186818), which enrolled 282 adults with symptomatic obstructive hypertrophic cardiomyopathy. The results are published in New England Journal of Medicine.
The study demonstrated that aficamten significantly improved peak oxygen uptake (peak VO₂), the trial’s primary endpoint, compared with placebo. Patients receiving aficamten also experienced improvements in symptoms and functional status, including better NYHA functional class, higher Kansas City Cardiomyopathy Questionnaire (KCCQ) scores, and significant reductions in left ventricular outflow tract (LVOT) gradients, indicating reduced obstruction to blood flow.
NICE concluded that aficamten is likely to provide clinical benefits comparable to mavacamten, the only other approved cardiac myosin inhibitor for obstructive HCM. The committee also noted that aficamten may require fewer echocardiographic assessments during dose titration, potentially reducing monitoring burden for patients and healthcare providers.
Safety and Monitoring
Because cardiac myosin inhibitors reduce heart muscle contractility, patients receiving aficamten require regular echocardiographic monitoring to assess left ventricular ejection fraction throughout treatment. According to the MHRA product information, commonly reported adverse reactions include dizziness and reversible reductions in left ventricular systolic function, making routine cardiac monitoring an essential component of therapy.
Regulatory Decision and NHS Access
NICE evaluated aficamten using a cost comparison approach, requiring the manufacturer to demonstrate that the medicine offers similar overall costs to the NHS as mavacamten. A confidential commercial arrangement was agreed between the manufacturer and NHS England to support routine NHS use.
The coordinated MHRA and NICE review enabled publication of final NICE guidance approximately two weeks earlier than the standard appraisal timeline. The initiative forms part of broader collaboration between the two organisations that has reduced NICE guidance timelines for medicines by around 30% since April 2024, allowing patients to access innovative therapies sooner following regulatory approval.
Under NHS funding rules, aficamten must be made routinely available across England within 30 days of NICE’s final guidance.
Regulatory and Patient Group Responses
Helen Knight, Director of Medicines Evaluation at NICE, said the recommendation demonstrates how closer collaboration between NICE and the MHRA can help patients gain faster access to clinically effective medicines while ensuring value for the NHS.
Julian Beach, MHRA Executive Director of Healthcare Quality and Access, said the coordinated decision highlights how regulatory innovation can accelerate patient access without compromising the agency’s rigorous standards for safety, quality and effectiveness.
Katharine McIntosh, Chief Executive of Cardiomyopathy UK, welcomed the availability of a second targeted treatment option, noting that a reduced need for echocardiographic monitoring during dose adjustment could improve convenience for both patients and healthcare services.
What This Means for Patients
The approval of aficamten provides adults with symptomatic obstructive hypertrophic cardiomyopathy whose symptoms remain despite standard treatment with a second targeted oral therapy alongside mavacamten. Beyond expanding treatment options, the coordinated MHRA-NICE review demonstrates a more efficient pathway for bringing innovative medicines into NHS practice by aligning regulatory approval and health technology assessment processes.
Patients and healthcare professionals are encouraged to report suspected adverse reactions through the MHRA’s Yellow Card Scheme, helping regulators continue to monitor the medicine’s safety following its introduction into clinical practice.
References
Thousands of patients to benefit from new daily pill for serious heart condition | NICE
About the Writer
Anamika Koshti (LinkedIn) is a PharmD professional and healthcare writer with interests in clinical research, pharmacovigilance, and evidence-based medicine. She has authored peer-reviewed publications on Alzheimer’s disease and PCOS, presented research at national conferences, and gained hands-on experience in medical content development and clinical data interpretation. She is committed to translating complex medical research into accurate, accessible content for healthcare professionals and patients.