The most expensive drug in the U.S. is now Lenmeldy, a $4.25 million gene therapy the FDA approved March 18 for children with a rare genetic disease, Becker Hospital Review reports.
Metachromatic leukodystrophy manifests into the loss of motor and cognitive function, and early death, according to the FDA. The first and only one-time medicine for patients is Lenmeldy (atidarsagene autotemcel). Its manufacturer, Orchard Therapeutics, said the drug’s wholesale acquisition cost is $4.25 million. In a March 20 news release, Orchard said the price tag ” is reflective of the value the therapy may deliver to eligible patients and their families, as well the potential long-term impact [the] treatment may have on overall healthcare utilization, minimization of productivity loss for caregivers and life opportunities for patients.”
In a trial, 37 children received Lenmeldy and experienced a significant reduction in the risk of severe motor impairment and death compared to untreated […]
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