A drug that treats the symptoms of a heart condition that can cause sudden death could offer hope to people with a family history of the condition.
The Scottish Medicines Consortium (SMC) has approved Camzyos (mavacamten) for use on the NHS in Scotland to treat obstructive hypertrophic cardiomyopathy. It follows a decision to sanction use of the medicine in England and Wales.
In obstructive HCM, the thickened part of the heart muscle, usually the wall (septum) between the two bottom chambers (ventricles), blocks or reduces the blood flow from the left ventricle to the aorta. Most people with HCM have this type.
It causes symptoms such as heart palpitations, chest pain, dizziness, fainting, shortness of breath, and fatigue.
HCM has been regarded as the most common cause of sudden cardiac death in young people and competitive athletes, although it is rare.
It is the condition that caused the-then 23-year-old footballer Fabrice Muamba to collapse during a game in 2012.
Doctors treating the player said his heart stopped beating for 78 minutes.
Mavacamten is the first drug to directly tackle one of the key disease processes that is thought to cause HCM.
Marc Dweck, Professor of Clinical Cardiology at the University of Edinburgh said: “A diagnosis of obstructive hypertrophic cardiomyopathy can have a major impact on quality of life for patients with this condition.
"The acceptance of mavacamten now allows eligible Scottish patients to access the first licensed treatment option to target an underlying cause of the disease, offering the potential for better symptom management and an improvement in quality of life.
"This new drug tackles a difficult problem," he added.
"In some patients with hypertrophic cardiomyopathy, the thickened heart muscle impedes the ejection of blood out of the heart.
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"Treatment options are limited and some patients still require major heart surgery.
"Mavacamten gives us another drug therapy for these patients that appears very effective in removing the obstruction and improving the ability of the heart to eject blood and pump it around the body.
He said further studies will examine if the drug could have a preventative role for people with a family history of the condition.
It’s estimated that 1 in every 500 people have HCM, but a large percentage of patients are undiagnosed.
Joel Rose, Chief Executive Officer, Cardiomyopathy UK (CMUK) said: “For people who experience symptoms of obstructive hypertrophic cardiomyopathy, everyday life can be impacted, and daily activities that we sometimes take for granted can become a challenge.
"Today’s news provides assurance that the latest advances are now accessible for those who need them.
"We believe that everyone affected by obstructive hypertrophic cardiomyopathy deserves to live well, and we hope that this treatment option supports eligible patients.”
Whilst some individuals with obstructive HCM may never experience symptoms, many others experience symptoms that can vary in severity.
Guy Oliver, general manager Bristol Myers Squibb, UK & Ireland said: "Today’s decision marks an important step towards ensuring equitable access to mavacamten in the UK. Now, patients across Scotland, England, Northern Ireland and Wales may benefit from this new treatment option.”
The SMC has also accepted treatments for severe alopecia, type 2 diabetes, chronic obstructive pulmonary disease and ulcerative colitis in its latest round of decisions.
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