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NHS offers the world's most expensive drug for children with a rare disease

Libmeldy is life-saving gene therapy now available for use in England


Key points from article :

Gene therapy Libmeldy is used to treat a rare condition, metachromatic leukodystrophy (MLD), caused by a crucial enzyme deficiency.

MLD develops in children younger than 30 months; lead to loss of sight, speech and hearing, as well as difficulty moving and seizures.

Around five babies are born with the disorder in England every year.

NHS chief executive Amanda Pritchard said: "This revolutionary drug is a life-saver for the babies and young children."

Libmeldy works by replacing the faulty gene that causes the disorder.

Long-term prognosis for children given the drug is still unknown.

Costing £2.8 million at its list price,it was the most expensive single treatment.

Orchard Therapeutics increased a confidential discount to the price the NHS actually pays.

The drug has been recommended for use by Nice in England only.

Patients from Scotland, Wales and Northern Ireland would need their national health systems to fund the treatment in Manchester.

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Amanda Pritchard

Chief Executive Officer of NHS England

National Institute for Health and Care Excellence (NICE)

This institution provides national guidance and advice to improve health and social care.


UK National Health Service, publicly funded healthcare system in England

Orchard Therapeutics

Company dedicated to transforming the lives of patients with rare diseases through innovative gene therapies

Topics mentioned on this page:
Gene Therapy, Nervous System