Key points from article :
After receiving FDA approval, Orchard Therapeutics has introduced Lenmeldy, a gene therapy for the rare disease metachromatic leukodystrophy (MLD), at a cost of $4.25 million.
This makes Lenmeldy the most expensive drug in the United States.
The Institute for Clinical and Economic Review (ICER), a U.S. drug cost watchdog group, supported Orchard's pricing after determining Lenmeldy's value-based price benchmark could reach up to $3.94 million.
Although Lenmeldy's cost is high, Orchard is working with payers (insurance companies, government programs) to create flexible payment plans that prioritize patient access.
Five medical centers in the U.S. will offer Lenmeldy treatment, with one in Minnesota already providing it in advance of full qualification.
MLD is a genetic condition affecting the brain and nervous system, and Lenmeldy works by inserting functional genes into a patient's stem cells.
