The FDA Approved The World’s Most Expensive Drug Last Week, A $4.25 Million One-Time Gene Treatment For An Ultra-Rare Nerve Disorder Typically Found In Toddlers

Gorodenkoff - - illustrative purposes only, not the actual people

In an era when groundbreaking medicines are being developed every day, a new drug has emerged as the world’s most expensive. The drug is a gene therapy for an ultra-rare disease, and it costs millions of dollars.

The one-time gene treatment, Lenmeldy, was approved by the U.S. Food and Drug Administration on March 18.

Orchard Therapeutics, the company that made it, announced that the drug will have a wholesale cost of $4.25 million, a price they claim to reflect the treatment’s value accurately.

Orchard focuses on research for gene therapies for rare diseases. The company is owned by the Japanese drugmaker Kyowa Kirin Co.

Lenmeldy will be used to treat metachromatic leukodystrophy (MLD), a nerve disorder typically found in toddlers. It causes them to lose the ability to walk and talk. Around half of the individuals with the hereditary condition die, while the others stay in a vegetative state.

MLD is also extremely rare, affecting about one in 100,000 live births and only around 40 kids a year in the U.S. The rarity of such diseases is what’s behind the high prices of gene therapies. Pharmaceutical companies say that they’re justified because they can save the lives of patients.

Orchard also has just 160 employees, which is much more than the number of kids they will treat even over several years. So, it makes sense why the new drug is so expensive.

But that just means that selling the DNA treatment could be a challenge since many of the people who need it won’t be able to get their hands on it.

Three years ago, the MLD therapy was approved in Europe, where the price was slightly lower. However, Orchard generated only $12.7 million from product sales last year, indicating that less than five kids got the treatment.

Gorodenkoff – – illustrative purposes only, not the actual people

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