Preventative Cure approved for Spinal Muscular Atrophy. Cost = 2.125M USD

[Teh_Diplomat 2,054]

 

https://www.theguardian.com/science/2019/may/25/21m-novartis-gene-therapy-to-become-worlds-most-expensive-drug

 

$2.1m Novartis gene therapy to become world's most expensive drug

 

 

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Swiss drugmaker Novartis has received US approval for its spinal muscular atrophy gene therapy Zolgensma – pricing the one-time treatment at a record $2.125m.

 

The Food and Drug Administration on Friday approved Zolgensma for children under the age of two with SMA, including those not yet showing symptoms. The approval covers babies with the deadliest form of the inherited disease as well as those with types where debilitating symptoms may set in later.

 

SMA is the leading genetic cause of death in infants. The disease often leads to paralysis, breathing difficulty and death within months for babies born with the most serious type 1 form. SMA affects about one in every 10,000 live births, with 50% to 70% having type 1. “This is potentially a new standard of care for babies with the most serious form of SMA,” said Dr Emmanuelle Tiongson, a Los Angeles paediatric neurologist who has provided Zolgensma to patients under an expanded access program.

 

Novartis executives have defended the price, saying a one-time treatment is more valuable than expensive long-term treatments that cost several hundred thousand dollars a year

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Zolgensma will compete with Biogen’s Spinraza, the first approved treatment for SMA.

 

 

Spinraza, approved in late 2016, requires infusion into the spinal canal every four months. Its list price of $750,000 for the initial year and $375,000 annually thereafter was also deemed excessive by Icer.

 

[DynamiteCop 2,088]

You watch Squirmy and Grubs huh?

 

 

[James Skywalker 528]

They're benign to the spine.

[Twinblade★ 3,986]

science wise this is good, as it certainly means that they're closer to developing treatments for diseases like ALS. But the price is still outrageous.

[DynamiteCop 2,088]

38 minutes ago, Twinblade said:

science wise this is good, as it certainly means that they're closer to developing treatments for diseases like ALS. But the price is still outrageous.

For now, it will come down eventually.