A ‘life-changing’ gene therapy for patients with haemophilia B is being made available on the NHS.
This breakthrough comes after years of research to progress new treatments.
It has the potential to significantly improve the lives of hundreds of people with the rare genetic bleeding disorder.
Patients at University Hospital Southampton (UHS) were among the first in the UK to receive the treatment.
They took part in a landmark clinical trial. Researchers found that a single injection of the gene therapy dramatically cuts the risk of bleeding.
Haemophilia patient John has welcomed the announcement as "fantastic" news.
‘A significant leap forward’
The National Institute for Health and Care Excellence (NICE) has approved the gene therapy Hemgenix for NHS use in England.
Around 2,000 people have haemophilia B in the UK. It is a rare genetic disorder that prevents the blood from clotting, due to a lack of clotting factor IX protein.
Even a small cut can be incredibly dangerous to someone living with the condition, as a wound will continue to bleed.
The lead for this research in Southampton is Dr Rashid Kazmi. He is a Consultant Haematologist at UHS.
“We are thrilled that gene therapy for haemophilia B will soon be available in England”, said Dr Kazmi.
“Hemgenix represents a significant leap forward in haemophilia B treatment. It is the only single-infusion gene therapy available in the UK that addresses the underlying cause of the condition.”
The treatment raises factor IX levels, keeps them high and reduces bleeding for years. This removes the need for regular injections.
It will be available for adults with moderately severe or severe haemophilia B in England.
Dr Kazmi continued: “This landmark development offers new hope to eligible patients, potentially freeing them from the burden of regular factor IX replacement therapy and improving their quality of life. It exemplifies our commitment to bringing cutting-edge treatments to those who need them most.
“We are proud to be at the forefront of this medical advancement. We look forward to seeing the positive impact it will have on the lives of haemophilia B patients across the country.”
Life-changing results
John Curley was one of just over 50 participants in the HOPE-B study.
He has not needed any treatment since receiving the gene therapy, which doctors describe as a ‘functional cure’.
John said: “It's fantastic that this treatment is now being made available to patients across England. Years ago, this could never have been dreamed of.”
In John’s childhood, he was restricted in playing any physical sports such as rugby or football.
Today, he enjoys playing football, riding his motorbike and is able to go on holiday without having to worry about insurance or travelling with large volumes of medication.
“It’s amazing to think that something I was told was incurable can now be effectively managed with a single-dose treatment,” John continued.
“It’s revolutionised my life physically and psychologically. I hope that haemophilia will be nothing but a distant memory in 30 years time.”
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