NHS watchdog approves ‘world’s most expensive drug’ that costs £2.6million a dose… and it will SAVE the health service money, say experts

A gene therapy once billed as the ‘world’s most expensive drug’ will be offered for free on the NHS, but experts insist it will save the health service money.

Called Hemgenix the drug costs £2.6million per patient, although only one dose is needed.

It is the only treatment of its kind for haemophilia B, a bleeding disorder where the body doesn’t make enough, or any, of a protein critical to clotting.

Clotting is a vital biological mechanism that stops wounds from bleeding meaning people with the disorder can suffer severe and even life-threatening blood loss if they suffer an injury.

Additionally, they also run the risk of what are called ‘spontaneous bleeds’ that occur without a direct injury and are potentially deadly if they occur in a vital organ.

Haemophilia B patients have to take frequent injections of an artificial clotting agent called Factor IX for life in order to keep their risk of catastrophic injury to a minimum.

But with Hemgenix the patient’s defective gene, which is incapable of producing the clotting, is replaced with one that can, eliminating the need for these injections.

Studies on the gene therapy, given via an IV drip, have shown the effects last for at least three years but the hope is it could work for even longer.

While there are approximately 2,000 people with haemophilia B in the UK, only 260 are estimated to be eligible for Hemgenix on the NHS.