Overview
- The Medicines and Healthcare products Regulatory Agency authorised aficamten on Wednesday 29 July and NICE published final guidance on Thursday 30 July that allows NHS England to make the drug available within 30 days.
- NICE recommended aficamten for adults with symptomatic obstructive HCM classified as New York Heart Association class II or III as an add‑on to optimised standard care or as a standalone option when those medicines cannot be used.
- Phase 3 trial data from SEQUOIA‑HCM showed patients on aficamten had a statistically significant improvement in exercise capacity and symptoms compared with placebo, supporting its approval.
- Common side effects reported include high blood pressure, palpitations, dizziness and systolic dysfunction, and the product is designated for additional monitoring with health professionals asked to report suspected reactions to the Yellow Card Scheme.
- About 1 in 500 people in the UK have hypertrophic cardiomyopathy, roughly half the cases are inherited, and NHS availability of a second oral myosin inhibitor may widen treatment choice and reduce early echocardiogram monitoring for some patients compared with the existing therapy.