Saturday 2 July 2022 11:21 PM Could haemophilia be 'cured' by a new gene therapy that costs £2.5million per ... trends now
A genetic drug hailed as 'close to a cure' for the deadly bleeding disorder haemophilia has been given the green light by European health watchdogs.
In trials, a single jab of Roctavian left nine in ten patients symptom-free and no longer in need of the daily injections usually required to control their condition.
Last week the European Medicines Agency, the equivalent of the UK's Medicines and Healthcare Products Regulatory Agency, approved the treatment, opening the door for its widespread use here.
But its price may push it out of reach for NHS patients: The Mail on Sunday has learned it could cost between £1.5 million and £2.5 million per dose, making it one of the most expensive medicines ever.
Dr Rashid Kazmi, consultant haematologist at University Hospital Southampton NHS Foundation Trust, says: 'When the latest Roctavian trial results were announced in March, we were euphoric.
A genetic drug hailed as 'close to a cure' for the deadly bleeding disorder haemophilia has been given the green light by European health watchdogs. (File image)
'For it to be approved in the UK, the price is obviously a concern. But haemophilia is a life-long condition, and treating it costs the NHS about £3,000 a month per patient.
'Over decades, this adds up. After a single jab of Roctavian, they can live life as normal.
'It's not a cure, as it doesn't work for everyone, but it's as close to a cure as we've got.'
Haemophilia is a rare disease caused by a genetic fault that means the body has problems producing a protein called factor VIII, which is needed for blood to clot.
The condition, which runs in families, is usually picked up during early childhood and about 8,700 people in the UK are currently living with it. In mild cases, sufferers still produce some factor VIII.
This means they have some ability to form blood clots and may simply experience nose bleeds and easy bruising.
But more than half of patients produce almost none, leaving them vulnerable to brain haemorrhages and spontaneous internal bleeding from their organs.
In trials, a single jab of Roctavian left nine in ten patients symptom-free and no longer in need of the daily injections usually required to control their condition. But its price may push it out of reach for NHS patients: The Mail on Sunday has learned it could cost between £1.5m and £2.5m per dose, making it one of the most expensive medicines ever
At present, sufferers keep problems at bay with injections of factor VIII. With Roctavian, also known as valoctocogene roxaparvovec, fragments of the missing genetic code needed to produce factor VIII are implanted into a harmless virus that is delivered into the patient via a drip.
The virus then penetrates the liver and transfers the genes, enabling the body to produce factor VIII for
