▶️ WATCH: The gene therapy Zolgensma has helped children born with a fatal disease, spinal muscular atrophy, grow up to run and play. But the cost is stunning: $2 million per dose. The drug’s price set the standard for other, even more expensive gene therapies. 📽️ : @josesepulveda.bsky.social - ThreadSky

propublica.org • 12 days ago

▶️ WATCH: The gene therapy Zolgensma has helped children born with a fatal disease, spinal muscular atrophy, grow up to run and play. But the cost is stunning: $2 million per dose. The drug’s price set the standard for other, even more expensive gene therapies.

📽️ : @josesepulveda.bsky.social

Comments

kaitensatsuma.bsky.social•12 days ago

I dunno what Jamiroquai was worried about

partylikeits1789.bsky.social•11 days ago

Anyone know who their CEO is and where they live? We should send them a letter.

indiaphelps.bsky.social•12 days ago

They are the robber barons of the 21st century

sindd.bsky.social•12 days ago

Greed is gross.

ysolde.bsky.social•12 days ago

They’re obscene.

childrensrights.bsky.social•12 days ago

For the benefits it provides, Zolgensma is worth every penny. But I hate that the pricing puts it out of reach for many patients.
Without the profit incentive, many of these drugs for rare conditions unfortunately would never see the light of day. The amount of profit is still gross, though.

batrachoid.bsky.social•12 days ago

Yeah, this is such an ugly situation. I know for a fact that even without the profit motive, these cures would still get researched by scientists, but it'd be absolutely impossible to get the kind of investor backing needed to turn them into actual usable therapies.

childrensrights.bsky.social•12 days ago

Vaccines have a similar problem, although they don't have the same "profit" problem :)

timothykolk.bsky.social•12 days ago

Ahem….EMINENT DOMAIN THE PATENTS.

kawon-zil.bsky.social•12 days ago

😳 $2 mil? 🫨😵‍💫

I haven't even read the article. Let me guess? U.S. taxpayers paid for a large portion of the research?

kaitensatsuma.bsky.social•12 days ago

Probably, but in this case it might be true that the technology behind making such changes is prohibitively expensive.

Or was, at least. Whatever happened to the potential of nanotechnology for this sort of stuff? 🤔

kawon-zil.bsky.social•12 days ago

But why should drug companies reap huge profits when taxpayers are the ones footing the bill? Before Congress changed the law, research like this belonged to everyone so drugs weren't insanely expensive. We should change the law back.

kaitensatsuma.bsky.social•12 days ago

That's a scientific question - the answer being is that genetic manipulation isn't a simple pill or drug you take once - but I'm rapidly getting out of my depth on this so we'll need to find someone with more intimate background in genetics to give more detail

kawon-zil.bsky.social•12 days ago

Read the article. This isn't about research. It's about price gouging (including by the researcher hired by the charity who turned around and made $400 million by selling the drug to a larger company). Taxpayers & the charity paid for the research. The drug co paid more to patent it than research.

kaitensatsuma.bsky.social•12 days ago

I didn't *say* it was about the research - but the article does reference that for about two dozen patients the company spent less than 12 million dollars so it's a pretty safe bet that the drug costs that much *at most* and costs significantly less now with dedicated production. Thank you

guillaume-deeptech.bsky.social•11 days ago

The way it works is that the returns for research bodies come from IP licensing. So Novartis is probably paying a substantial amount of money to use this IP, and the research body probably had a stake in AveXis. At least they had all the power to negociate this, as is routinely done for spinoffs.

propublica.org•12 days ago

Yep, you're right. Taxpayers and charities subsidized much of the science that led to Zolgensma. Here's the full article:

kawon-zil.bsky.social•12 days ago

"Kaspar alone took in more than $400 million.Kaspar alone took in more than $400 million..."
👿👿👿

kawon-zil.bsky.social•12 days ago

"The drug’s trials were small, often involving two dozen patients or fewer. AveXis, and later Novartis, spent less than $12 million up to the point of the drug’s approval — surprisingly little — to prove the therapy was safe and effective..."
😡😡😡

kawon-zil.bsky.social•12 days ago

Sophie Gaynor and her parents are true American heroes.

kawon-zil.bsky.social•12 days ago

OMG... only a few paragraphs in and my blood is boiling!!!

cylindricalsea.bsky.social•12 days ago

I have a feeling the number of hoops to jump through (by the patient) to get their "health" insurance company to approve a treatment is equivalent to the number of dollars each dose costs.

sfthugwife.bsky.social•12 days ago

My daughter died of this in 2013 at 7 weeks. If it had been around I think we might have gone bankrupt just to give her a chance but what a terrible predicament for parents.

patient23.bsky.social•12 days ago

Novartis should be ashamed of themselves!

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