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RandomFactor writes:

The Food and Drug Administration has a approved a new gene therapy from Novartis that rings in at a whopping $2.125 Million for a one-time treatment making it "the most expensive drug on the market."

The drug Zolgensma is the second gene therapy approved for treatment of a genetic disease and consists of an infusion of genetically modified viruses carrying healthy copies of a defective gene that causes spinal muscular atrophy (SMA), which can be fatal within a year or two of birth. The first, approved in 2017 for treatment of a genetic form of blindness, was priced at a comparatively modest $425,000 per eye.

These backbreaking (eye watering?) prices come with their share of controversy

"We have been slowly subjected to price increases the same way the frog in the boiling water is slowly boiled to death," [Peter Bach, MD, MAPP Director of the Memorial Sloan Kettering's Center for Health Policy and Outcomes says.]

Insurers are expected to cover the cost. The company says payment plans will be available.

AveXis president Lennon acknowledges the numbers might seem shocking. But he argues the drug is easily worth it. The only existing treatment for spinal muscular atrophy, a drug called Spinraza, costs hundreds of thousands of dollars a year. Zolgensma hopefully will be a one-time, life-saving treatment.

"We're talking about a lifetime of benefit being condensed down into a one-time treatment," Lennon says. "We're not used to thinking about this that way. We're used to a system of a chronic medication where we spread things out over years if not decades."

The drug is currently in production and will be available for use "shortly"

Side Note: The fable of the frog being slowly boiled alive is doubly inaccurate. Frogs will progressively become more active attempting to escape slowly heating water and if able will exit the 'pot' long before it comes to the point they are in danger. Additionally a frog dropped into boiling water would likely be unable to hop out in time to save itself.

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