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Following US FDA approval of Lenmeldy™ (atidarsagene autotemcel) for early-onset metachromatic leukodystrophy (MLD), the US wholesale acquisition cost of the gene therapy has been set to $4.25 million.
The new Clinical Biotechnology Centre (CBC) in Bristol will manufacture cell and gene therapies to help boost the UK life sciences sector.
The one-time gene therapy treatment for rare, neurodegenerative disease metachromatic leukodystrophy (MLD) is the most expensive drug ever evaluated by NICE.
In this article, we summarise the trial findings for the European Medicines Agency’s 13 most transformative therapies of 2020.
Ten medicines have been recommended for marketing authorisation and 10 extensions of indication have been suggested by the EMA.
