A first-of-its-kind treatment targeting a chronic heart disease could offer a “greater hope” to thousands of people living with the condition.
The National Institute for Health and Care Excellence (Nice) has approved the use of mavacamten in draft guidance to the NHS. It would be used to treat those with obstructive hypertrophic cardiomyopathy (HCM), with about 7,000 people expected to benefit.
The drug, also known as Camzyos, is the first to specifically target the chronic disease, which in 50% of cases is caused by an inherited genetic mutation. It would be prescribed alongside other medicines such as beta blockers, non-dihydropyridine calcium channel blockers or disopyramide.
Those with obstructive HCM have excessive heart muscle contractions, which cause the organ to become thickened and stiff and stop it from pumping sufficient blood to meet the body’s needs.
Symptoms include tiredness, dizziness, shortness of breath, chest pains and heart palpitations, and it can lead to complications such as an irregular heartbeat, heart failure, stroke and sudden cardiac death.
In its draft guidance, Nice said clinical trials suggested that mavacamten plus standard care was more effective than standard care alone and could delay the need for surgery.
Helen Knight, the director of medicines evaluation at Nice, said the move was “another example” of the organisation’s “commitment to drive early access to innovative new treatments”.
Mavacamten is yet to be licensed for use in the UK and Nice will not publish its final guidance on the drug until it has been approved.
Knight added: “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.”
Prof Sir Nilesh Samani, the medical director at the British Heart Foundation, said: “This is very welcome news for patients with obstructive HCM, who often have debilitating symptoms that are not improved by current treatments. Mavacamten provides a real breakthrough, and it is good to see that its approval has been fast-tracked by Nice so patients can benefit from an improved quality of life sooner.”
