WIREDGOV

NICE has approved mavacamten, the first treatment that specifically targets a heart condition called obstructive hypertrophic cardiomyopathy (HCM). It means around 7,000 people will now be able to access the treatment on the NHS.

NICE’s final draft guidance recommends mavacamten (also called Camzyos and made by Bristol-Myers Squibb) as an add-on to standard care that aims to control symptoms of the disease.

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

Obstructive HCM is a chronic disease which in 50% of cases is caused by an inherited genetic mutation. It is characterised by excessive heart muscle contraction which causes it to become thickened and stiff. As a result, the heart may not be able to pump enough blood to meet the body’s needs.

Obstructive HCM can cause debilitating symptoms including tiredness, dizziness, shortness of breath, chest pain and palpitations. This can have a severe and wide-ranging impact on quality of life.

It can also lead to a range of complications including arrhythmias (irregular heartbeat), heart failure, stroke and sudden cardiac death. Some people with uncontrolled symptoms may choose to have surgery.

Helen Knight, director of medicines evaluation at NICE, said:

“Today’s draft guidance is another example of our commitment to drive early access to innovative new treatments – in this case a treatment which is yet to be licensed for use in the UK.

“Obstructive hypertrophic cardiomyopathy is a disease for which, until now, there has been no specific treatment that targets its underlying cause. It has a very high and wide-ranging impact on quality of life and because it can develop at any age, it can occur in younger people who may formerly have had very active lifestyles.

“The treatments currently used to try to manage symptoms are associated with side effects and are often ineffective. We’re therefore pleased to be able to recommend a treatment that has the potential to alter the course of obstructive hypertrophic cardiomyopathy and offer greater hope to people with it.”

The draft guidance recommends mavacamten for adults with HCM whose symptoms mean ordinary physical activity results in fatigue, palpitations or shortness of breath.