Mavacamten for Obstructive HCM Approved for Scotland

News 08 April 2024

Mavacamten, the first ever drug specifically for a type of cardiomyopathy, has been approved for use in Scotland. This follows its consideration by the Scottish Medicines Consortium (SMC). The SMC decides which evidence-based treatments are available through the NHS in Scotland.

The drug will be made available as an option for treating obstructive hypertrophic cardiomyopathy (HCM) in adults, as an add-on to standard care. This means that mavacamten will be available to Scottish patients who are struggling with their obstructive HCM despite taking the drugs that are currently recommended. It will also be available to people for whom existing treatments are not suitable. You can read the recommendation in full here and a simple overview here

The SMC’s recommendation echoes that of NICE (the National Institute for Health and Clinical Excellence). NICE approved mavacamten for use in the rest of the UK last year.

In clinical trials, Mavacamten was effective in relieving obstruction, controlling symptoms and improving quality of life for people with obstructive HCM. For some people the drug removed the need for more invasive surgery such as a myectomy.

Next Steps

It may take a few months before people in Scotland who are eligible for this treatment receive it. This is because hospitals need to have the the medicine in stock and systems in place to make sure it is delivered safely. We will make sure that the new treatment can reach people who may be eligible as quickly as possible. We will continue talking to our network of healthcare professionals in Scotland and to the company that developed Mavacamten (Bristol Myers Squibb).

If you have obstructive HCM, please speak to your doctor about whether this new drug is suitable for you.

Our role

SMC operates in a similar way to NICE and decides whether to recommend a drug becomes available on the NHS. It makes this decision based on evidence that it receives from the drug’s manufacturer, health care professionals, clinical experts and others, including charities. This means that we were invited to be part of the decision-making process.

Over the last year, we collected evidence from our national survey, special focus groups, and from our helpline. This meant that when we presented our evidence to the SMC, we were able to give an honest account of the impact that obstructive HCM has. We described how it affects an individual’s physical and mental health, as well as the strain it can put on the whole family. We were able to “put a face on the condition” and show the difference that Mavacamten could make. 

Cost considerations are an important part of the SMC’s decision-making. They have to consider whether the NHS can afford new medicines. So we also spoke to Bristol Myers Squibb about the importance of putting the drug at a reasonable price.

Cardiomyopathy UK CEO Joel Rose said:

"This is a significant step forward for the cardiomyopathy community in Scotland. It brings Scotland in line with the rest of the UK. Cardiomyopathy has a huge impact on peoples’ physical and mental health. It makes day-to-day living a real struggle. Mavacamten has proven to be effective in relieving symptoms for people with obstructive HCM. We hope that it is the first of many new and effective treatments for people with all forms of cardiomyopathy."

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