Libmeldy®, an autologous gene therapy, has been used for the first time on the NHS

Libmeldy® is a treatment for early-onset metachromatic leukodystrophy (MLD), a rare, inherited and otherwise fatal disease that causes progressive demyelination and neurodegeneration. The treatment involves extracting the patient’s stem cells, inserting a working copy of the genes back into the cells, and administering the corrected genes via IV infusion. This one off treatment is hoped to cure the patient completely.

The breakthrough treatment has been used for the first time in treating a 19-month old toddler, Teddi Shaw. Teddi received treatment between June and October 2022. The list price of the treatment is £2.8 million per patient. It is understood that the NHS and the manufacturer, Orchard Therapeutics, agreed to a significant discount. The news is a reminder of the ongoing tension between the amazing potential of cell gene therapy on the one hand, and the pricing challenges which pose obstacles to access. There are currently only five European treatment centres for Libmeldy®.

For more insight on licensing considerations with cell gene therapy, see my colleague, Louisa Jacobs’, post on "Cell and gene therapies - licensing considerations" here.