The ‘world’s most expensive drug’ is set to become available on the NHS for children in England that need it, it has been announced.
For children suffering from a rare, fatal genetic condition, they will require access to a specific drug that happens to be the most expensive form of medicine in the world. This will now be made available through NHS services in England alone.
The change in accessibility comes after NHS bosses negotiated a “significant” price cut from the drugs original value that is being kept confidential.
What is the ‘world’s most expensive drug’?
Libmeldy is a single-dose gene therapy treatment that is used to treat metachromatic leukodystrophy (MLD).
The drug goes by atidarsagene autotemcel and it is sold under libmeldy which is created by UK-based pharmaceutical firm Orchard Therapeutics.
The drug removes the patient’s stem cells to replace the faulty gene from the deadly condition before re-injecting the treated cells into the patient. Libmeldy prevents the development of an enzyme that builds up the fats that destroy the protective layers in children’s nerves.
MLD is a rare and genetic condition that causes severe harm to a child’s nervous system and organs, which can cause a life expectancy to as low as five to eight years.
How much does libmeldy cost?
Libmeldy is priced at £2.8million – which is the most expensive drug in the world.
Now, NHS bosses have negotiated a price cut from this original value that is said to be “significant” but the amount is staying confidential.
The increased accessibility to this vital drug will be beneficial in saving children’s lives who have MLD. MLD can develop in babies younger than 30 months, leading to loss of sight, speech and hearing, as well as cognitive problems, brain impairment, seizures and also death.
When and where will libmeldy be available from the NHS?
The gene therapy treatment will be available from five different European sites with only one being based in the UK.
The UK facility serving the libmeldy will be Manchester’s Center for Genomic Medicine at Saint Mary’s Hospital.
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.”