1 Recommendations

1.1 Mavacamten is recommended as an option for treating symptomatic obstructive hypertrophic cardiomyopathy in adults who have a New York Heart Association class of 2 to 3. It is recommended only if:

  • it is an add‑on to individually optimised standard care that includes beta‑blockers, non-dihydropyridine calcium-channel blockers or disopyramide, unless these are contraindicated, and

  • the company provides it according to the commercial arrangement.

1.2 This recommendation is not intended to affect treatment with mavacamten that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop.

Why the committee made these recommendations

Treatment for obstructive hypertrophic cardiomyopathy aims to manage its symptoms. Standard care is either beta-blockers or non-dihydropyridine calcium-channel blockers and if symptoms persist then disopyramide may be added. Some people with uncontrolled symptoms may choose to have surgery. Mavacamten is the first treatment that specifically treats the condition rather than the symptoms. For this evaluation, the company asked for mavacamten to be considered only as an add-on treatment for people having optimised standard care. This is a narrower population that does not include everyone covered by mavacamten's marketing authorisation.

Clinical trial evidence suggests that mavacamten plus standard care is more effective than standard care alone, and that it may avoid or delay the need for invasive surgery.

The most likely cost-effectiveness estimate for mavacamten is within the range that NICE considers an acceptable use of NHS resources. So mavacamten is recommended.