UK greenlights aficamten: thousands with obstructive HCM gain faster access to once-daily therapy

England is set to offer a new oral treatment to an estimated 6,600 adults living with obstructive hypertrophic cardiomyopathy after regulators and health economists moved in tandem to clear the drug for NHS use. The move follows simultaneous marketing authorisation from the MHRA and a positive recommendation from NICE, paving the way for rapid rollout across the country.

What this approval means for patients?

Obstructive hypertrophic cardiomyopathy thickens and stiffens the heart muscle, limiting blood flow and triggering symptoms such as fatigue, shortness of breath, dizziness, chest discomfort and irregular or rapid heartbeats. Roughly one in 500 people in the UK has some form of HCM, and about half of cases run in families.

Aficamten, sold under the brand name Myqorzo, is formulated as a once-daily tablet. It belongs to a class called cardiac myosin inhibitors, which work by calming excessive contraction in heart muscle cells. This helps the heart pump more efficiently, eases obstruction to blood flow and can improve day-to-day symptoms for eligible adults with NYHA class II to III disease.

Clinical studies, including the phase 3 SEQUOIA-HCM trial, show that aficamten can boost exercise capacity and reduce symptom burden in obstructive HCM. Evidence suggests it performs on par with mavacamten, the only other NICE-backed therapy for this condition, while potentially requiring fewer dose-finding scans during treatment setup.

Faster pathway to NHS availability?

NICE applied a cost-comparison framework for this appraisal. To secure a positive recommendation, the sponsor had to show that aficamten's costs are comparable to or lower than those of mavacamten, which is already recommended for symptomatic obstructive HCM. A confidential discount has been agreed to support NHS affordability.

Thanks to tighter coordination between MHRA and NICE, final guidance was issued two weeks sooner than usual. Broader reforms have already accelerated NICE timelines by around 30 percent for medicines and 9 percent for health technologies compared with pre-2024 processes.

NHS England is expected to make aficamten available within 30 days of the final NICE guidance publication.

How aficamten works?

The active ingredient, aficamten, reversibly binds to myosin in cardiomyocytes, reducing hypercontractility that drives left ventricular outflow tract obstruction. Patients will undergo periodic heart function checks, including echocardiograms, to ensure safe and effective dosing.