CNN Health reports that a new gene therapy for metachromatic leukodystrophy is the world's most expensive drug.
The US Food and Drug Administration has recently approved the drug, called Lenmeldy, which is marketed in Europe as Libmeldy, for the treatment of the fatal genetic disorder.
The drug's manufacturer announced a wholesale cost of $4.25 million.
The NHS approved Libmeldy in 2022 for some patients with metachromatic leukodystrophy (see BioNews 1132), with a list price of £2.8 million.
Since then, the first NHS recipient of the drug in the UK, has been cured of the disease (see BioNews 1179).
