NHS deal for ‘most expensive drug’ to treat a rare disease that affects children

BABIES AND children with a rare genetic disease have been given new hope after the NHS has struck a deal for the drug to treat it – reportedly the most expensive medication in the world.

Libmeldy is used to treat metachromatic leukodystrophy (MLD), an illness which attacks the nervous system and organs. Most children diagnosed with MLD have a life expectancy of just five to eight years.

Libmeldy has a reported list price of £2.8m but the NHS has negotiated a significant discount, it revealed yesterday, meaning it will be free at the point of use for children in England. Libmeldy works by removing the patient’s stem cells and replacing the faulty gene that causes MLD before then reinjecting the treated cells into the patient.

The most common form of MLD 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. Around four babies are born every year with the condition.

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 cuttingedge 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.”

The rollout of the treatment is to begin in the spring at Manchester’s Centre for Genomic Medicine, one of just five European sites that will administer the treatment.