Babies with a Fatal Genetic Disease will now be able to access the world’s most expensive drug costing up to £2.8million on the NHS.
The health service has secured a confidential discount on the list price, allowing doctors to offer young children the life-saving treatment from spring.
The revolutionary gene therapy, called Libmeldy, gives patients the prospect of leading a normal life, health bosses say.
It is used to treat metachromatic leukodystrophy (MLD), which usually develops in babies younger than 30 months and causes severe damage to the nervous system and organs.
MLD results in a life expectancy of between five and eight years.
Babies with a fatal genetic disease will now be able to access the world’s most expensive drug costing up to £2.8million on the NHS
It is estimated around four babies born every year in England will have the condition and Previous Treatment Options were limited to managing symptoms and supportive care.
The drug will be available at Manchester’s Centre for Genomic Medicine at Saint Mary’s Hospital. It will be the only site in the UK – and just one of five in Europe – to offer the treatment.
It has a list price of more than £2.8 million, making it the most expensive drug in the world.
But it can now be offered to young patients on the NHS in England after the health service negotiated a ‘significant’ confidential discount.
Libmeldy a one-time therapy intended to correct the underlying cause of MLD – a faulty gene whose dysfunction leads to a build up destructive fat around the child’s nerves.
Scientists obtain stem cells from the patient and remove the faulty gene before re-injecting the treated cells back into the body.
Amanda Pritchard, chief executive of NHS England, 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.’
Amanda Pritchard, chief executive of NHS England, said the ‘revolutionary drug’ is a life-saver for the babies and young children who suffer from metachromatic leukodystrophy (MLD)
Previous treatment options for MLD were limited to managing symptoms and supportive care.
It is estimated that around four babies born every year in England will have the condition.
Professor Simon Jones, Consultant in Paediatric Inherited Metabolic Disease at Manchester University NHS Foundation Trust, said: ‘This is enormously welcome news for all families affected by MLD, a progressive, life-limiting condition for which there were previously no approved treatment options.’
