Or­chard sets US price tag for rare dis­ease gene ther­a­py Len­meldy at $4.25M

Or­chard Ther­a­peu­tics’ ul­tra-rare dis­ease drug Len­meldy is vault­ing to the list of most ex­pen­sive drugs ever with a whole­sale ac­qui­si­tion cost of $4.25 mil­lion for the one-time treat­ment in the US.

The com­pa­ny an­nounced the price Wednes­day fol­low­ing Len­meldy’s FDA ap­proval ear­li­er this week, say­ing that it re­flects “its clin­i­cal, eco­nom­ic and so­ci­etal val­ue.”

Len­meldy is the on­ly FDA-ap­proved treat­ment for chil­dren with ear­ly-on­set metachro­mat­ic leukody­s­tro­phy, a rare neu­rometa­bol­ic dis­ease that af­fects an es­ti­mat­ed few­er than 40 chil­dren an­nu­al­ly in the US, ac­cord­ing to Or­chard. 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. Lib­meldy has al­ready gen­er­at­ed $12.7 mil­lion in sales in the first nine months of 2023, ac­cord­ing to Or­chard’s last earn­ings re­port.

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