New drug offers hope for a rare form of heart strain

Lifesaving new drug offers hope to 7,000 Britons a year who suffer a dangerous form of heart strain

  • Mavacamtem is the first drug to treat obstructive hypertrophic cardiomyopathy 
  • It causes heart muscle to thicken and stiffen – making it hard to pump blood

Thousands of people with a potentially deadly heart condition are set to benefit after NHS bosses sanctioned use of a groundbreaking new drug.

Mavacamten – also known as Camzyos – is the first medicine developed specifically to tackle obstructive hypertrophic cardiomyopathy, a serious disorder that affects about 7,000 people in the UK.

It causes heart muscle to thicken and become stiff – making it harder for it to pump oxygen-rich blood round the body. Symptoms include severe fatigue, dizziness, shortness of breath, chest pain and palpitations.

About 50 per cent of cases are genetic and there is a high risk of heart failure, stroke and even sudden death from the heart stopping completely.

Doctors mainly treat it with drugs that control blood pressure and ease the strain on the heart, but many still get little relief.

Mavacamten – also known as Camzyos – is the first medicine developed specifically to tackle obstructive hypertrophic cardiomyopathy, a serious disorder that affects about 7,000 people in the UK

About 50 per cent of cases are genetic and there is a high risk of heart failure, stroke and even sudden death from the heart stopping completely

Trials of Mavacamten showed that – when given alongside existing medicines – it eased tiredness, palpitations and breathlessness. Some patients improved so much that they were able to avoid open-heart surgery, which would trim away excess cardiac muscle and improve the heart’s function.

Now the National Institute for Health and Care Excellence (NICE), the NHS drug spending watchdog, has issued guidance calling on doctors to prescribe it when patients are still feeling poorly despite being on existing medications.

Helen Knight, director of medicines evaluation at NICE, said: ‘This condition can occur at any age, even in younger people with very active lifestyles. And, until now, there has been no specific treatment that targets the underlying cause. This drug has the potential to alter the course of the disease and offers greater hope to people with it.’

Joel Rose, chief executive of heart muscle charity Cardiomyopathy UK, welcomed the move. ‘This is a significant step forward for people with cardiomyopathy,’ he said.

‘We hope it’s the first of many new and effective treatments.’

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