In a move greatly welcomed by heart charities, the National Institute for Health and Care Excellence (NICE) has approved mavacamten for treating symptomatic obstructive hypertrophic cardiomyopathy (HCM) in adults.
NICE said that the drug was "the first treatment that specifically treats the condition rather than the symptoms" and that its approval meant that around 7000 people will now be able to access the drug on the NHS.
In final draft guidance, NICE approved mavacamten as an option for adults with HCM with a New York Heart Association class of 2 to 3 whose symptoms meant that ordinary physical activity resulted in fatigue, palpitations, or shortness of breath.
The regulator said that mavacamten (Camzyos, Bristol-Myers Squibb) was recommended to be made available as an add-on to individually optimised standard care that aimed to control symptoms of the disease and included beta-blockers, non-dihydropyridine calcium-channel blockers, or disopyramide, unless these were contraindicated.
The drug was recommended as long as the company provided it according to a commercial arrangement with a confidential discount. Mavacamten does not yet have marketing authorisation in Great Britain and its list price is commercial in confidence until this is granted.
Drug Could 'Avoid or Postpone' the Need for Surgery
Clinical trial evidence suggested that mavacamten plus standard care was more effective than standard care alone, and that adding the drug could "avoid or postpone the need for invasive surgery", NICE said.
Helen Knight, director of medicines evaluation at NICE, said: "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.
Current Treatments 'Often Ineffective'
"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."
Patient support charity Cardiomyopathy UK, which was one of the groups submitting evidence to NICE's committee, tweeted that the recommendation was a "big step forward" for the cardiomyopathy drug. Greeting the announcement, it said : "People who are struggling with their obstructive HCM despite trying the drugs that are currently recommended, or people for whom existing drugs are not appropriate, should now be able to access this new medicine."
In clinical trials, mavacamten was "effective in relieving obstruction, controlling symptoms, and improving quality of life", the charity said. "For some people the drug removed the need for more invasive surgery such as a myectomy."
However, it noted that, although the NICE recommendation was "a significant step forward", there were "a few more stages to go through before the drug can be made available". In particular, it needed to receive marketing authorisation from the Medicines and Healthcare Products Regulatory Agency (MHRA), which was anticipated within the next few months.
Hospitals Must Have Systems in Place to Make Mavacamten Available
When NICE recommends a treatment 'as an option', the NHS must make sure it is available within a set period for patients who meet the criteria and whose treating clinician thinks that it is the right treatment.
Once the drug receives its licence, therefore, "hospitals will need to have their systems in place to be able to order sufficient supplies and closely monitor those people who are taking the drug," Cardiomyopathy UK alerted. CEO Joel Rose said: "This is a significant step forward for the cardiomyopathy community.
"Cardiomyopathy has a huge impact on peoples' physical and mental health, making day to day living a real struggle. Mavacamten has proven to be effective in relieving symptoms for people with obstructive HCM and we hope that it is the first of many new and effective treatments for people with all forms of cardiomyopathy."
Mavacamten a 'Real Breakthrough'
Asked to comment by Medscape News UK, Professor Sir Nilesh Samani, 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.”
NICE's final guidance is expected to be published on 12 July 2023.