Costly Drug for Fatal Muscular Disease Wins F.D.A. Approval


***The Food and Drug Administration has approved the first drug to treat patients with spinal muscular atrophy, a savage disease that, in its most severe form, kills infants before they turn 2.

The drug, called Spinraza, will not come cheap — and, by some estimates, will be among the most expensive drugs in the world.***

Read the rest here:

This is huge. If a child receives the treatment before displaying symptoms, it is effectively a cure. They never will lose their ability to walk. And even for those who already have displayed symptoms, the drug has been shown to increase muscle function.

But how many will actually be able to receive it? The article goes on to report that one year of treatment with Spinraza will cost $750,000!

It seems so wrong to charge that amount.


Very interesting article, Robyn! A single dose injection costs $125,000, according to the article. This kind of profiteering on human misery is crazy, but I’m sure the pharmaceutical company will simply counter that they spent enormous amounts of money researching it and testing it and getting it approved, and so their price is justified.

This argument is currently much talked about in Washington state, because recently a very, very expensive cure for Hepatitis C was made available to all Hepatitis C patients. It’s legally mandatory for physicians to supply the drug to any Washington resident who’s afflicted with the disease, regardless of income or lack of health insurance. Meaning, this expensive drug called Harvoni, which costs $1000 per pill and which has a very, very high cure rate, has to be given to anyone with Hepatitis C. Which is great, because Hepatitis C is a deadly disease, and a huge global epidemic. So the pharmaceutical profiteering is now the problem of the state government, not individuals. And by the way, anyone reading this who has friends or relatives with this illness, you might take this into consideration. Establishing residency in Washington state to be treated and cured, might well be worth it.

I don’t want to hijack your thread by the way, Robyn. It just seems like this is exactly the same kind of thing. I’m pretty sure the way these ludicrous medicine prices are overcome is by people who have the disease filing lawsuits against both the pharmaceutical company, as well as against there own health insurance company. Because if this form of muscular dystrophy is fatal, then it would seem to be out-and-out murder to withhold the medicine because someone’s too poor to pay for it. I’m sure that’s how it will unfold.


Spinraza treats a genetic disorder that affects just 9,000 people. Rare-disease drugs have so far been insulated from pricing controversies because without FDA orphan designation status there’d be no drugs.


I think if I were in that position I would try to emigrate from the U.S.


Not hijacking at all! I do hope a similar law can be passed for Spinraza. These kids’ lives are at stake.

What makes me a little angry here is Spinraza was funded by the SMA community. They’ve spent decades raising the money for research and now that there is finally a result, the company is turning around and charging them $125,000 per dose? After all the support they gave? I get that the drug is probably expensive to produce but come on. No one can possibly pay that.

Going to pray that something changes and people are able to get the treatment they deserve. :gopray:


I get that, but it still is infuriating.

Also, that number is a bit misleading. Only 9,000 people in the US currently are diagnosed. Meaning they are living with the disease. But the disease affects something like 1 in every 6000 infants. The problem is most of them don’t survive past toddler age, so that’s why the number seems so low.

If a law was passed that a) required a test at birth for the SMA gene and b) guaranteed treatment for any infant diagnosed (like the Hep C drug Exiled Child mentioned) then theoretically no child would ever be killed from the disease again. Hopefully this will happen.


What I have read and seen before is usually that when a person has insurance, the insurance company will be charged full price. When the person has no insurance, the company usually hands out some sort of coupon that will allow the person who needs an expensive orphan drug will be charged something like $.01 per pill or even get it for free.

There is a lot of profanity (almost solid profanity in the beginning) in the following video, so be warned, but it gets the above point across: [FULL]

Martin Shkreli raised the price of an orphan drug (daraprim) which helped AIDS patients from a very low price to something like $750 per pill.

If you can stand a fair amount of profanity and nonsense, you can learn quite a bit about the way things actually work in the orphan drug industry in comparison to the way the media portrays how things work by listening to Shkreli’s videos.


It will be interesting to learn how other countries handle the high price of this drug. I suspect they will not pay the high cost, and still have good health outcomes for these patients. That is my guess.

That is one item I read, it is difficult to figure out drug pricing. I remember reading a former New England Journal of Medicine’s editors book on that topic, about how drug are priced. As she found, how drugs are priced is a closely held secrete. Most drug development costs come form tax payers. It is typically later, when a drug is nearly ready for marketing that drug firms become involved.

Dr.Marcia Angell’s book can be seen here:

“The Truth About the Drug Companies: How They Deceive Us and What to Do About It”


My granddaughter has to have an injection which costs $4000 a month. (She only has to take this for another year or two). Of course her parents can’t pay the full price, but my daughter
was able to deal directly with the pharmaceutical company and they supply many doses for free. My husband takes a very expensive drug that is not covered well by insurance, and he gets it directly off the pharmaceutical company for about $35.00 a month. Many companies have an address or number to call if you can’t afford a medication and often will help, and there may be a state agency that will help.

But i don’t know about help for a drug that costs 3/4 of a million dollars a year!


This thread seems related to the recent passage of the bipartisan 21st Century Cures bill; in part, it speeds up the FDA approval process. It sounds like a measure to be thankful for and be very cautious of at the same time.

If we see more deregulation in the near future (which I would assume to be the case) we’ll be seeing more treatments available - especially for crippling and terminal illnesses. Yet, costs will probably be enormous. I’m not sure how much liability insurance is buried in costs but it may play a part.


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