We are delighted to announce that the Scottish Medicines Consortium (SMC) has recently undertaken its final review of atidarsagene autotemcel (Libmeldy®), a life-changing gene therapy for treating Metachromatic Leukodystrophy (MLD), and has given a positive recommendation for its use within NHS Scotland.
Children eligible are:
children with late infantile or early juvenile forms, without clinical manifestations of the condition.
children with the early juvenile form, with early clinical manifestations, who still have the ability to walk independently and before the onset of cognitive decline.
SMC reported that after considering all the available evidence and the output from the Patient and Clinician Engagement (PACE) process, they accepted atidarsagene autotemcel for use in NHS Scotland. They confirmed that:
"the Committee considered the benefits of atidarsagene autotemcel in the context of the SMC decision modifiers that can be applied when encountering high cost-effectiveness ratios and agreed that as atidarsagene autotemcel is an ultra-orphan medicine, SMC can accept greater uncertainty in the economic case."
Sophie Thomas, Interim Chief Executive of the MPS Society, said: "Today’s decision by the SMC to recommend Libmeldy® is a significant milestone for the MLD community and a powerful example of what can be achieved through collaboration.
"We are incredibly grateful to the patient organisations, families, clinicians and partners who worked together to ensure the voices and experiences of patients, parents and carers were heard throughout this process."
The final recommendation can be found here.
More information
Pharma company details
Orchard Therapeutic’s press release
About the patient organisations
MPS Society
Contact: Sophie Thomas (Interim CEO)
mpssociety.org.uk 0345 389 9901
Alex TLC
alextlc.org 020 7701 4388
MLD Support Association UK
mldsupportuk.org.uk 07414 529392
ArchAngel MLD Trust
archangel.org.uk
You can find out more about atidarsagene autotemcel (brand name: Libmeldy®) in the Patient Information Leaflet (PIL) by searching for the medicine name on the Medicines and Healthcare products Regulatory Agency (MHRA) website.
https://products.mhra.gov.uk/
