Mavacamten – the first treatment for obstructive hypertrophic cardiomyopathy (HOCM) that specifically treats the condition rather than its symptoms – has been recommended for use on the NHS in England and Wales since 2023 and in Scotland since 2024. For many people who have been prescribed it, the results have been remarkable. But research undertaken by Cardiomyopathy UK suggests that many people with HOCM who could benefit from the drug aren’t getting it.
This article explores our key findings. While they’re not representative of everybody potentially eligible for mavacamten, they provide insights into views and experiences.
Click here to read the report in full
A potentially life-changing treatment
23% of the 116 people with HOCM who took part in our initial survey had been prescribed mavacamten. Of these, 76% said they were satisfied or somewhat satisfied by their experience.
The comments we received revealed that, for some people, mavacamten was “transformational” and “life-changing” – with energy returning, symptoms like dizziness and palpitations resolving, and a more normal day-to-day life restored.
These are exactly the kinds of outcomes that make mavacamten such a significant treatment for people living with HOCM – but challenges remain around access to the drug on the NHS.
Access challenges
As mentioned, 23% of the people with HOCM who took part in our initial survey had been prescribed mavacamten. Given mavacamten's eligibility is determined by specific criteria, a significant portion of patients may not meet the requirements for it to be prescribed – however, this figure falls well below the expected 50% of ‘real-world HOCM patients’ that are likely to be eligible.
Several respondents reported long waiting times to get onto mavacamten. Some also noted that living at a distance from a prescribing centre can be a significant obstacle. This is especially the case because mavacamten requires regular monitoring via ECGs and echocardiograms. While some people find the monitoring regime reassuring, some find it frustrating.
Adding to this, there are still areas of the UK with no mavacamten prescribing service at all.
Awareness is another issue. Nearly half of those who hadn’t seen a consultant for 12 months or more, or who had but hadn't discussed mavacamten, said they were unaware of the drug before taking our survey – so they wouldn’t have been able to ask about it themselves.
NICE (the National Institute for Health and Care Excellence) is currently assessing aficamten, another drug for treating HOCM. Some of the access challenges associated with mavacamten – including the intensity of the monitoring schedule – are anticipated to be less of an issue with aficamten. We submitted evidence to that appraisal and we’ll share more as NICE guidance develops.
To inform our submission, we carried out a separate survey earlier this year on the experiences of people living with HOCM, capturing an up-to-date snapshot of their care and treatment on the NHS. This research similarly highlighted the positive impact mavacamten had on symptoms for many respondents, alongside mixed experiences around appointments. Travel distance was a recurring theme, with several respondents reporting long journeys which, when combined with waiting times and tests, could amount to several hours away from home.
Our recommendations
Our survey results make it clear that, while mavacamten can have a real impact on symptoms, systemic challenges mean that not enough people with HOCM are accessing it.
Based on our findings, we're calling on:
- consultant cardiologists to discuss mavacamten with patients where appropriate
- hospital trusts to improve referral pathways and establish prescribing centres where none yet exist
- NHS England and the Department of Health and Social Care to ensure newly approved medicines reach patients faster.
We're also committing to step up our own awareness-raising work, so that more people with HOCM know about mavacamten and feel equipped to have informed conversations with their care team.
Click here to read the report in full
Advice and support if you need it
If you have HOCM and you’d like to find out more about mavacamten and your treatment options, speak to your specialist nurse or cardiologist. You can also speak to our specialist nurse team for general advice and support about cardiomyopathy. You can call our free helpline on 0800 018 1024 (Monday to Friday, 8.30am to 4.30pm) or email supportnurse@cardiomyopathy.org.