The World’s Most Expensive Drug Is Now a $4.25 Million Gene Therapy

A new gene therapy for an ultra-rare disease will have a wholesale cost of $4.25 million, making it the world’s most expensive drug.

The one-time treatment, Lenmeldy, won U.S. regulatory approval on Monday to correct the underlying cause of a hereditary condition called early-onset metachromatic leukodystrophy, or MLD.

MLD is a fatal disease in which infants sometimes start to lose the ability to walk and talk. Orchard Therapeutics said the drug’s price “reflects its clinical, economic and societal value” in a statement Wednesday. 

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The drug’s cost tops that of CSL Behring LLC’s Hemgenix, a one-time infusion for hemophilia priced at $3.5 million.

MLD affects about one in 100,000 live births, and fewer than 40 children a year in the U.S., according to Orchard.

The company focuses on developing gene therapies, which aim to correct the underlying genetic flaws that cause inherited diseases. It was recently acquired by the Japanese drugmaker Kyowa Kirin Co. 

The field of gene therapy has sparked debate over its high prices. Pharma companies say they are justified because the treatments can potentially cure patients, generating savings for the Health-care system over time and delivering other societal benefits. 

Drugmakers also want to recover the cost of developing treatments that are often aimed at small populations, limiting their revenue potential. But pricing has raised concern about the strain it could put on insurers, particularly Medicaid, the Health-insurance program for the poor.