NICE recommends Mavacamten for Obstructive HCM in new draft guidance

News 01 June 2023

NICE (National Institute for Health and Clinical Excellence), the organisation that decides which evidence based treatments are available through the NHS, has published their final draft guidance on Mavacamten, the first ever drug specifically for cardiomyopathy.

They have recommended that the drug is made available as an option for treating symptomatic (New York Heart Association class 2 to 3) obstructive hypertrophic cardiomyopathy (HCM) in adults. It is recommended only if it is an add-on to individually optimised standard care including beta-blockers, non-dihydropyridine calcium channel blockers or disopyramide, unless these are contraindicated.

This means that 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. You can read the draft guidance in full here

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.

NICE’s recommendations only cover England, Wales and Northern Ireland. It is likely that the Scottish Medicines Consortium (SMC) will meet later this year to consider if the drug should be made available in Scotland.

Next Steps

Receiving NICE recommendation is a significant step forward but there are a few more stages to go through before the drug can be made available.

As well as being recommended for use in the NHS by NICE, drugs also need to receive marketing authorisation, often called a licence, from the Medicines and Healthcare Products Regulatory Agency (MHRA). This is to ensure drug safety and quality. It is expected that Mavacamten will receive its licence in the next few months. After that, 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.

We are talking to the company that developed Mavacamten (Bristol Myers Squibb) and our network of healthcare professionals to get a better understanding of when people with obstructive HCM may start to receive the drug. At this stage we believe that the drug should be available by the end of this year, but this may change, and we will keep you updated.

Our role

NICE decides whether to recommend a drug based on evidence that it receives from the drug’s manufacturer, health care professionals, clinical experts and other stakeholders including charities. This means that we were invited to be part of the decision-making process by NICE.

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 NICE, we were able to give an honest account of the impact that obstructive HCM has on 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. We also spoke to Bristol Myers Squibb about the importance of making the drug affordable to the NHS.

Laura Kelly, who shared her own experience of living with obstructive HCM with the NICE committee that reviewed Mavacamten, said:

"I used to live life to the full. I was fit, active and having fun. But Obstructive HCM robbed me of my previous life. Having a chance to share my story was really important to me. I wanted people to see the real-life impact of obstructive HCM, what it has done to me and to my family. The decision to recommend Mavacamten is just fantastic news, and I am delighted to have played a part in it."

Cardiomyopathy UK 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."

All documents relating to NICE's appraisal of Mavacamten can be found here

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