The gene therapy treatment is used to treat metachromatic leukodystrophy (MLD), which causes severe damage to a child’s nervous system and organs
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A life-saving treatment touted as the most expensive drug in the world is being rolled out by the NHS for babies and young children suffering from a rare and fatal genetic disease.
The gene therapy treatment, known by its brand name Libmeldy, is used to treat metachromatic leukodystrophy (MLD), which causes severe damage to a child’s nervous system and organs.
Those suffering from the disease typically have a life expectancy of between five and eight years.
The single-dose treatment has a reported list price of more than £2.8 million, which would make it the most expensive drug in the world.
However, the treatment can now be offered to young patients in England after the health service said it negotiated a “significant confidential discount”.
Libmeldy, manufactured by UK-based pharmaceutical firm Orchard Therapeutics, works by removing the patient’s stem cells and replacing the faulty gene that causes MLD before re-injecting the treated cells into the patient.
The NHS said it prevents the development of a crucial enzyme that leads to a build-up of fats, which destroy the protective layers around the child’s nerves.
The most common form of MLD usually develops in babies younger than 30 months and can lead to loss of sight, speech and hearing, as well as difficulty moving, brain impairment, seizures, and eventually death, the health service said.
It is estimated that around four babies born every year in England will have the condition.
The treatment will be available on the NHS from Manchester’s Center for Genomic Medicine at Saint Mary’s Hospital – one of just five European sites that will administer the treatment, and the only site in the UK.
The previous most expensive drug was Zolgensma, a one-off gene therapy to treat Spinal Muscular Atrophy (SMA) in babies and young children, which had a reported list price of £1.79 million per dose.
NHS chief executive Amanda Pritchard said: “This revolutionary drug is a life-saver for the babies and young children who suffer from this devastating hereditary disorder and will spare their families untold heartache and grief.
“The deal we have struck is just the latest example of NHS England using its commercial capabilities to make good on the NHS Long Term Plan commitment to provide patients with cutting-edge treatments and therapies at a price that is fair to taxpayers.
“It also shows that while rolling out the world-leading NHS Covid vaccination programme, and caring for people with the virus, the health service is also doing its very best to care for millions of patients with other conditions.”