A Lifesaving Therapy for Children with a Rare Disease Is Now the World’s Most Expensive Drug, Raising Questions About Access

March 20, 2024

3D rendering of a DNA molecule

(CNN) – 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 Food and Drug Administration 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. (Read More)