FDA Approves Revolutionary $4.25 Million Gene Therapy for Rare Genetic Disorder

Lenmeldy becomes the most expensive drug globally, offering hope for children with metachromatic leukodystrophy.

A new gene therapy named Lenmeldy has been approved by the U.S. Food and Drug Administration for treating metachromatic leukodystrophy (MLD), an ultra-rare and fatal genetic disorder.
Lenmeldy is now the world's most expensive drug, with a wholesale cost of $4.25 million for a one-time treatment.
The therapy is designed to correct the underlying genetic cause of MLD by adding functional copies of the ARSA gene to a patient's own blood stem cells.
Clinical trials have shown that Lenmeldy significantly reduces severe motor impairment or death in children with early-onset MLD, with promising long-term outcomes.
Concerns have been raised about the affordability and insurance coverage for this costly treatment, despite its potential to save lives and reduce long-term healthcare costs.