NHS to roll out new gene therapy for metachromatic leukodystrophy

NHS to roll out new gene therapy for metachromatic leukodystrophy
© iStock/Jacek_Sopotnicki

The NHS has agreed on a deal for life-saving treatment for metachromatic leukodystrophy that will offer babies and young children the prospect of a normal life.

The revolutionary gene therapy treatment named Libmeldy is used to treat metachromatic leukodystrophy is reportedly the most expensive drug in the world. Metachromatic leukodystrophy causes severe damage to the child’s nervous system and organs, resulting in a life expectancy of between five and eight years.

This deal is part of the NHS Long Term Plan, a plan drawn up by frontline staff, patient groups, and national experts to create a service fit for the future. The aim is to provide cutting-edge treatments and therapies to patients.

Libmeldy: the world’s most expensive drug?

Libmeldy has a reported list price of more than £2.8 million, making it the most expensive drug in the world. It will now be available to young patients on the NHS in England after the health service negotiated a confidential discount.

Libmeldy works by removing the patients stem cells and replacing the faulty gene that causes metachromatic leukodystrophy before re-injecting the treated cells into the patients. It prevents the development of a crucial enzyme that leads to a build-up of fats that destroy the protective layers around the child’s nerves.

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.”

Metachromatic leukodystrophy: A rare and fatal genetic disease

The most common form of metachromatic leukodystrophy 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.

Previous treatment options for metachromatic leukodystrophy were limited to managing symptoms and supportive care. It is estimated that around four babies born every year in England will have the condition.

The drug, which has been manufactured by UK-based pharmaceutical firm Orchard Therapeutics, has been recommended for use by the National Institute for Health and Care Excellence (NICE) today (Friday) in its draft guidance. NICE will shortly publish final guidance on the use of Libmeldy, enabling the rollout of the treatment to begin in the Spring. It is planned that it will be delivered by a specialist service and is available on the NHS from Manchester’s Centre for Genomic Medicine at Saint Mary’s Hospital. This is one of just five European sites that will administer the treatment, and the only site in the UK.

Professor Simon Jones, Consultant in Paediatric Inherited Metabolic Disease at Manchester University NHS Foundation Trust (MFT), 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. While there are various sub-types of the condition, in its later stages, all forms largely result in children losing their ability to move and speak.

“It is extremely pleasing that this treatment will now be offered to all eligible patients, which is testament to the rigorous clinical trials that have paved the way for this decision, and the world-leading research and innovation capability within the NHS.

“As a Trust, we are delighted to be selected as one of five European centres to begin offering this new service as part of NHS standard of care, furthering our work as a European centre of excellence for metabolic conditions.”

Clinical trials have shown this treatment will deliver substantial health benefits, drastically improving quality of life, elongating life expectancy and the prospect of a normal life. The treatment will be available to babies and young children with no clinical signs or symptoms, as well as those with early symptoms of the condition, so long as they can still walk independently and with no evidence of cognitive decline.

Where there is no family history of the condition, MLD is usually diagnosed through a combination of brain MRI and blood and urine tests before genetic testing is used to identify the specific mutation.

Professor Bobby Gaspar, MD, PhD, chief executive officer of Orchard Therapeutics, said: “Today’s landmark agreement with NHS England follows a thoughtful and comprehensive value assessment by NICE and represents a major milestone for the MLD community, Orchard, and the entire field of hematopoietic stem cell gene therapy.

“We are delighted that NICE and NHS England have recognized the transformational clinical impact of Libmeldy for eligible MLD patients, and thank the leading clinicians and advocates involved for their tireless engagement throughout the process. A deep body of evidence now points to the potential for durable effects in HSC gene therapy for certain severe genetic diseases including MLD.”


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