Libmeldy (atidarsagene autotemcel) was approved by the European Commission to treat metachromatic leukodystrophy (MLD) in December 2020, and subsequently rejected for NHS use by NICE last July on the grounds that it was too expensive – even at the discount offered at the time – and had uncertain long-term benefits.
A second confidential price reduction means that children in England and Wales with MLD will now be able to get treatment with the one-shot therapy, which has a list price of £2.8 million.
