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European health authorities have given the green light to a genetic drug that is said to be “almost curable” for the deadly bleeding disorder hemophilia.
In studies, a single Roctavian shot resulted in nine out of 10 patients remaining symptom-free and no longer requiring the daily injections normally required to control their condition.
Last week, the European Medicines Agency, the equivalent of Britain's Medicines and Healthcare products Regulatory Agency, approved the treatment, paving the way for widespread use here.
But its price could make it unaffordable 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 drugs ever.
Dr. Rashid Kazmi, consultant haematologist at University Hospital Southampton NHS Foundation Trust, said: “When the latest results from the Roctavian trial were announced in March, we were ecstatic.
European health authorities have given the green light to a genetic drug that is said to be “almost curable” for the deadly bleeding disorder hemophilia. (file image)
"Price is obviously an issue for approval in the UK. But haemophilia is a lifelong condition and its treatment costs the NHS around £3,000 per month per patient.
"That adds up over decades. After a single shot of Roctavian, they can lead their lives as normal.
“It’s not a cure because it doesn’t work for everyone, but it’s as close to a cure as we have.”
Hemophilia is a rare disease caused by a genetic error that means the body has problems producing a protein called factor VIII, which is needed for blood clotting.
The condition, which runs in families, is usually identified in early childhood and around 8,700 people in the UK are currently living with it. In mild cases, those affected still produce some factor VIII.
This means that they have some ability to form blood clots and can easily get nosebleeds and easy bruising.
But more than half of patients produce almost none, making them vulnerable to brain hemorrhages and spontaneous internal bleeding from their organs.
In studies, a single Roctavian shot resulted in nine out of 10 patients remaining symptom-free and no longer requiring the daily injections normally required to control their condition. But its price could make it unaffordable 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 drugs ever
Currently, those affected are keeping problems at bay with factor VIII injections. Roctavian, also known as valoctocogene roxaparvovec, involves implanting fragments of the missing genetic code needed to produce factor VIII into a harmless virus that is given to the patient via a drip.
The virus then enters the liver and transfers the genes so that the body can produce factor VIII itself.
In the most recent study, 134 patients received a Roctavian infusion. After one year, nine out of ten patients were effectively cured - with normal or near-normal factor VIII levels. After two years, some had relapsed, but 75 percent were still either mildly affected or completely normal.
Side effects included nausea and headaches, while 86 percent of patients experienced liver inflammation requiring oral steroids.
Another disadvantage is that Roctavian is a one-time treatment, and should a patient suffer a relapse, a second dose cannot be given.
Because the drug uses a virus to deliver the genetic material to the liver, the body's immune system produces antibodies that would attack and destroy it if it were to enter the body again.
According to industry reports, pharmaceutical company BioMarin, which makes Roctavian, has said it will cost between £1.5 million and £2.5 million per dose.
Experts agreed it was likely to initially be listed at that price, but the NHS medicines regulator, the National Institute for Health and Care Excellence, is expected to negotiate a significant discount.
Despite the high price, Dr. Kazmi: “As there is so much data on the safety and use of the drug, I think it will be available in the UK within a year.”
Patient Jamie Murray, 23, from Glasgow, was treated with Roctavian in September 2019 and has since been healthy and no longer needs medication, despite suffering from a severe form of the disease throughout his life.
The gaffer was diagnosed at 18 months old and received factor VIII injections every other day throughout his childhood.
Because of his condition, he has never played contact sports, had to constantly carry extra medication and inject himself with extra injections before doing anything that caused bleeding, such as going to the dentist.
He says: "When I was a child there were often tears when it came to my injections, but as I got older I didn't know any different."
In September 2019, Jamie enrolled in a Roctavian trial and received an infusion of the drug at University Hospital Southampton.
He then continued his regular factor VIII medication until the levels in his blood began to rise.
He says: "Within a month or so I got to the point where the doctor said if I had a serious accident it would affect me no differently than anyone else as my factor VIII levels are the same as a normal person."
Jamie has not had a bleed since October 2019 and three years later his factor VIII levels remain well within the normal range.
Despite suffering from mild liver inflammation for which he was taking steroid tablets, he says: “I have never regretted taking the drug - it never crossed my mind.
“I hope more people get access to it as it has changed my life.”