An Update on Mavacamten

Latest Research 25 May 2022

In February 2021 we shared the news that a US pharmaceutical company, MyoKardia, had announced promising results from their EXPLORER-HCM clinical trial of the drug Mavacamten. Mavacamten is the first drug developed to target a specific molecular defect that causes obstructive hypertrophic cardiomyopathy (HCM). The trial confirmed the drug’s ability to reduce obstruction, control symptoms and improve quality of life for people with obstructive HCM. Since then, the company that developed the drug has been taken over by Bristol Myers Squibb (BMS), a large international pharmaceutical company.  

At the end of April 2022, the US Food and Drug Administration gave approval for the drug (known under the brand names “Camzyos”) to be used in the treatment of adults with symptomatic obstructive HCM. The drug was to be used to improve functional capacity and symptoms.  

Here in the UK, the National Institute of Health and Care Excellence (NICE) will decide whether the drug should be made available on the NHS. NICE will be reviewing evidence towards the end of 2022. Cardiomyopathy UK are part of that review process. We will be presenting the views and experiences of people with obstructive HCM to the NICE team to provide a true picture of the impact of the condition and the needs of people affected by it. 

We have already begun to gather our evidence. Last month we held a focus group for people with obstructive HCM. The people we spoke to all felt that it was important to consider the impact of the condition on their emotional wellbeing. For some this was because their condition meant that they had to limit their physical activity, something that they really enjoyed previously. The group all felt that they would want to see alternative and less invasive treatments than ablation or myectomy to alleviate their symptoms. They also worried about the long-term impact of their current medication. We will continue to collect evidence and in the next few months we will be sending out a national survey to gather further insight for NICE.  

It is too early to say if or when Mavacamten will be available to people in the UK, but we are in regular contact with NICE and with the pharmaceutical company, BMS, to monitor the situation. We will do everything we can to give a true and meaningful account of the difference that this new treatment could have on peoples’ lives. 

If you have obstructive hypertrophic cardiomyopathy and would like to take part in our national patient survey that will inform the NICE review, please email to learn more. 


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