NICE approves first gene therapy for neurodegenerative disorder affecting children

NICE has approved a new therapy for use in the NHS in children with Metachromatic Leukodystrophy (MLD), a rare neurodegenerative genetic disorder.

MLD is a progressive disorder which first starts to appear in childhood, and eventually means that patients lose their ability to move or speak. Around four children a year in England are born with the condition.

The new therapy, atidarsagene autotemcel (with licensing name Libmeldy), was developed by Orchard Therapeutics – a UK-based pharmaceutical company – and has a list price of £2.8 million. Libmeldy therefore ousts Zolgensma as the most expensive drug licensed under the NHS. Zolgensma which was licensed for use in infants with spinal muscular atrophy by the NHS in March 2021, has a list price of £1.8 million (see BioNews 1098).

'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' said Amanda Pritchard, the chief executive of NHS England. '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'.

MLD is caused by a genetic variant, and the disease progression reduces the nerve cells' ability to transmit signals from the brain. There are different forms of MLD that all ultimately cause difficulty with talking and eating, requiring support such as feeding tubes. Libdelmy has been approved for those with late infantile onset, when onset occurs before 30 months of age, and juvenile onset, between 30 months and six years of age, which currently have typical life-expectancies of five to eight and ten to 20 years respectively. Trials of the treatment showed that it significantly increased quality of life and life expectancy, and might offer the prospect of a normal life.

The therapy works by taking stem cells from the patient's own bone marrow or blood, and using a lentivirus vector to insert functional copies of the faulty gene into the patient's own stem cells. These are then injected back into the patient as a single dose IV treatment, following a single round of chemotherapy to kill the stem cells containing the faulty gene.

Just five sites in Europe will offer the treatment, and it will be offered in the UK by the Centre for Genomic Medicine at Saint Mary's hospital in Manchester. Professor Simon Jones, a consultant at Manchester University NHS Foundation Trust said: 'These kinds of therapies are going to be expensive, they are going to be hard to deliver and only a few centres are going to be able to do that.

'There are probably quite a few different disorders which can be treated with this approach, but Libmeldy is the first to really break ground.'