The one-time treatment, Lenmeldy, which is a new gene therapy for an ultra-rare disease for children will cost $4.25 million, making it the world’s most expensive drug in the world.
Recently Lenmeldy was approved by the US Food and Drug Administration (FDA) on Monday. The first gene therapy for metachromatic leukodystrophy (MLD) for a rare and fatal disease called early-onset metachromatic leukodystrophy, or MLD primarily affecting children under the age of seven.
It is a fatal disease in which infants sometimes start to lose the ability to walk and talk. Orchard Therapeutics the company that makes Lenmeldy said in a statement Wednesday ( March 27) the drug’s price “reflects its clinical, economic and societal value.”
According to Orchard, the disease MLD affects about one in 100,000 live births, and fewer than 40 children a year in the U.S.
The company (Orchard Therapeutics) focuses on developing gene therapies, which aim to correct the underlying genetic flaws that cause inherited diseases. Recently the company was acquired by the Japanese drugmaker Kyowa Kirin Co.
However, The wholesale cost for this lifesaving therapy 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.
