The NHS can now offer a "transformative" new treatment to patients with a rare blood disorder after the national medicines watchdog gave funding approval.
Around 200 people with haemophilia B will be eligible for the Hemgenix gene therapy drug, which helps the body produce blood-clotting factors and "frees" users from the need for regular treatments, said Bloomberg.
At £2.6 million a dose, the therapy is "one of the world's most expensive" treatments. The National Institute for Health and Care Excellence (Nice) has endorsed the investment for "a limited period" while Hemgenix's efficacy "remains under scrutiny".
What is haemophilia B? Due to a DNA mutation, patients with haemophilia B either can't produce enough of factor IX – a specific protein that makes blood clot – or lack it entirely. "Without this crucial clotting component, bleeds are bigger and longer," said the BBC. Much rarer than haemophilia A, around 2,000 people in the UK have the condition.
Treating haemophilia B requires constant care and monitoring. People with the blood disorder receive weekly injections of factor IX, with extra injections needed if a patient injures themselves or undergoes surgery.
How does this gene therapy work? Hemgenix is administered as a "one-off infusion, lasting about an hour", said the BBC. Patients receive "engineered viruses" with "copies of the fully functional factor IX instructions". The viruses "act like a fleet of microscopic postmen, delivering those blueprints to the liver", which can follow the instructions to start producing the clotting protein.
What impact will the treatment have? Professor Stephen Powis, NHS England's national medical director, said the therapy could be "truly life-changing".
Elliott Collins, who took part in a clinical trial of the treatment in 2019, told the BBC that "I feel cured". Before, "I would have to think about it all the time", he said, so "for it to completely disappear" was to feel changed "mentally and physically".
The therapy may not be effective for all patients. Of 54 men who took part in the clinical trial, two continued to need factor IX injections. And for those for whom the treatment was effective, "nobody knows how long it will last". But some research has suggested Hemgenix could sustain factor IX levels for at least 10 years.
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