FDA greenlights Orchard’s gene therapy Lenmeldy for rare and fatal disease

The FDA ap­proved a gene ther­a­py known as Len­meldy for ear­ly-on­set metachro­mat­ic leukody­s­tro­phy, or MLD, a rare and fa­tal ge­net­ic dis­ease that leads the ner­vous sys­tem to stop func­tion­ing over time.

The reg­u­la­tor on Mon­day an­nounced the ap­proval for Or­chard Ther­a­peu­tics. The gene ther­a­py was ap­proved as Lib­meldy in the EU in 2020 and in the UK in 2022, where it has a list price of £2.8 mil­lion. Or­chard CEO Bob­by Gas­par told End­points News on Mon­day ahead of the an­nounce­ment that the US list price would be an­nounced soon af­ter the ap­proval.

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