Orchard Therapeutics’ gene therapy Libmeldy (atidarsagene autotemcel), reportedly the most expensive drug in the world, is to be made available on the National Health Service (NHS) in England for some children with the neurodegenerative condition metachromatic leukodystrophy (MLD), NHS England has announced. The news comes after the health service negotiated a “significant confidential discount” to the price of the drug, which has a list price of more than £2.8 million.
The announcement coincides with the publication of new draft guidance from the National Institute for Health and Care Excellence (NICE) recommending the use of Libmeldy, the first ever treatment for MLD. The one-off treatment will be available to babies and young children with no clinical signs or symptoms of MLD, as well as those with early symptoms of the condition provided that they can still walk independently and with no evidence of cognitive decline. Previous draft guidance from NICE issued in July 2021 had recommended against use of Libmeldy on the grounds that it was too expensive and had uncertain long-term benefits. But, after a public consultation the manufacturer increased the confidential discount to the price of the drug.
Commenting, Helen Knight, programme director in the Centre for Health Technology Evaluation at NICE said, “The clinical evidence presented showed that for those children in whom atidarsagene autotemcel was effective, it had a substantial clinical benefit in terms of enabling children to maintain their motor and cognitive functions. We’re therefore pleased that the company has agreed an improved discount to the price of atidarsagene autotemcel which, in better reflecting the uncertainty in the evidence, has enabled the committee to recommend this potentially transformative treatment for children with this condition”.
Roll-out of the treatment will begin in spring 2022 once NICE has issued its final guidance. The therapy will be available through the Royal Manchester Children’s Hospital in collaboration with Manchester’s Centre for Genomic Medicine at Saint Mary’s Hospital, the only site in the UK and one of only five European sites that will administer the treatment.
It is estimated that there are four to five children born with MLD each year in England.
NHS Chief Executive Amanda Pritchard states, “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”.
www.england.nhs.uk, “NHS to roll out life-saving gene therapy for rare disease affecting babies”, 4th February 2022
www.pharmaphorum, “NHS signs deal with Orchard for pricey Libmeldy gene therapy”, 4th February 2022
www.nice.org.uk, “First gene therapy for rare genetic neurodegenerative disorder in children, recommended in NICE draft guidance”, 4th February 2022
www.bbc.co.uk, “Libmeldy: World’s ‘most expensive’ drug recommended for NHS use”, 4th February 2022