A transformational gene therapy which “cures” the bleeding disorder haemophilia B has been made available on the NHS.
The National Institute for Health and Care Excellence (Nice) has recommended the therapy, which is reported to be one of the world’s most expensive medicines.
It involves a one-time infusion that allows patients with severe haemophilia to stop regular treatments for a limited period while the drug’s effectiveness is scrutinised.
A commercial agreement was signed with its manufacturer, CSL Behring, and it will be the first treatment to enter NHS England’s innovative medicines fund (IMF).
The treatment costs £2.6 million per patient and through the IMF, NHS England will immediately fund the treatment at eight specialist centres, including in Birmingham, Manchester and Oxford.
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Haemophilia B is a genetic blood-clotting condition, caused by a deficiency of Factor IX, a naturally occurring protein essential for blood coagulation, resulting in either a partial or complete lack of its activity.
An estimated 2,000 people in the UK have the condition, which primarily affects men, causing prolonged or spontaneous bleeding, often in joints, muscles, and internal organs.
Etranacogene dezaparvovec is the active substance in the branded Hemgenix gene therapy drug and uses a modified virus that contains copies of the gene responsible for producing Factor IX.
The patient is given the virus to deliver a healthy Factor IX gene to the liver cells, enabling them to produce the missing protein, thereby limiting the bleeding episodes.
It is given as a one-off infusion that takes one to two hours, in addition to reduced frequency of the routine Factor IX protein injections that many patients with severe haemophilia B have to endure on a weekly or bi-weekly basis.
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The treatment focuses on reducing spontaneous and traumatic bleeds, enabling physical activity and preventing joint damage.
Hemgenix will be available on prescription through the NHS as a treatment option for adults with moderately severe or severe haemophilia B. An estimated 250 patients will be eligible.
Elliott Collins, from Colchester, Essex, spent 29 years of his life needing regular injections to help clot his blood. Since taking part in a clinical trial for gene therapy five years ago in 2019, he has not needed any further treatment and feels “cured”.
Collins, who is now 34, spent his childhood rebelling against his diagnosis by playing rugby and skateboarding, but any injury risked a damaging bleed, especially when he was tackled. He told Today on BBC Radio 4: “I got studs across the shin and that came up like a cartoon, like Tom and Jerry.”
Some trials have suggested that the treatment may last for more than a decade, but more research is needed to confirm whether the price tag is worth the benefit.
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The weekly injections that are routinely prescribed on the NHS, cost between £150,000 and £200,000 per patient annually, while Hemgenix has an official list price of £2.6 million.
Collins is already preparing himself for the effects to wear off. “I’m very aware of the fact that this is new, I’m almost preparing myself for it to potentially wear off to avoid any kind of major disappointment,” he said.
Responding to final draft guidance from Nice, Professor Sir Stephen Powis, the national medical director of NHS England, said: “This promising drug is the latest in a series of pioneering gene therapies secured for NHS patients at an affordable price and becomes the first drug to be made available in our innovative medicines fund to provide early access for patients while further data is collected on its long-term benefits.”
