A lifesaving therapy for children with a rare disease is now the world’s most expensive drug, raising questions about access

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.

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. The wholesale cost isn’t usually what patients pay, but it’s a cost that’s considered and shouldered by public and private health insurance plans, including state Medicaid plans, which cover roughly 4 out of every 10 children in the United States. Manufacturers of gene therapies say the big prices reflect big benefits — the chance to be free of a disabling or even fatal disease — and they point out that they need to be able to recoup the steep costs of development, testing and manufacturing their products. Health policy experts say that as the list of gene and cell therapies with eye-popping prices grows, it may strain the ability of states and other insurers to cover their costs, and ultimately limit patient access if plans begin to exclude these therapies as a class from coverage. Dr. Bobby Gaspar, the co-founder and CEO of Orchard Therapeutics, the company that makes Lenmeldy, said the treatment is “paradigm-shifting medicine and has the potential to stop or slow the progression of this devastating childhood disease with a single treatment.”