Posted by Michael Wonder on 20 Mar 2024
A lifesaving therapy for children with a rare disease is now the world’s most expensive drug, raising questions about access
20 March 2024 - A new gene therapy for the fatal genetic disorder metachromatic leukodystrophy, or MLD, will carry a wholesale price of $4.25 million, its manufacturer announced Wednesday, making it the world’s most expensive medicine.
Lenmeldy was approved by the US FDA on Monday and is the first therapy for the rare and devastating disease, which typically kills affected children before they turn 7. About 40 children are born with MLD in the US each year.
