¡The most expensive treatment in the world! - Health Communications Ltd

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The most expensive treatment in the world

September 17 2024

Zolgensma, The single-dose treatment for babies born with a rare and often fatal genetic disease (spinal muscular atrophy – SMA) was introduced to the market in 2019 at the exorbitant cost of $2.1 million per unit. However, things have changed.

In 2020, the FDA approved ridisplam, sold as Evrysdi, for older children and adults with spinal muscular atrophy (SMA). The approval has recently been expanded to include infants younger than two months, making it a more direct rival to Zolgensma.

Other gene therapy developers are pouring billions into new drugs. There are more than 1,500 ongoing clinical trials of cell and gene therapies registered with U.S. regulators, and last year the FDA approved five, including two for sickle cell disease that had higher list prices than Zolgensma: $2.2 million and $3.1 million.

¡However, things are always changing!

There’s a new drug that’s more expensive than ever: a gene therapy that costs as much as a brownstone in Brooklyn or a mansion in Miami, and far more than the average person will earn in a lifetime.

Lenmeldy is a genetic treatment for metachromatic leukodystrophy (MLD) and was approved in the U.S. this year. Its manufacturer, Orchard Therapeutics, says the wholesale cost is $4.25 million, slightly more for patients and families, or for the government to foot the bill.

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