The Lenmeldy treatment is used to correct the cause of early-onset metachromatic leukodystrophy.
Drugmakers want to recover the cost of developing treatments that are often aimed at small populations, limiting their revenue potential.
The one-time treatment, Lenmeldy, won US regulatory approval on March 18 to correct the underlying cause of a hereditary condition called early-onset metachromatic leukodystrophy, or MLD. The drug’s cost tops that of CSL Behring’s Hemgenix, a one-time infusion for haemophilia priced at US$3.5 million.
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