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posted by martyb on Thursday September 22 2016, @12:05AM   Printer-friendly [Skip to comment(s)]
from the false-sense-of-hope dept.

Eteplirsen received approval for use as a Duchenne muscular dystrophy therapy despite the FDA review team concluding that the treatment was unlikely to show any benefit for patients.

Dr. Janet Woodcock's (Director of the Center for Drug Evaluation and Research) decision was heavily influenced by the "parading diseased children in front of the cameras" and was made before the FDA's review team completed their analysis.

Part of Dr. Woodcock's rational for approval included the stock price of Sarepta (the pharmaceutical company responsible for eteplirsen):

She opined that Sarepta in particular "needed to be capitalized." She noted that [Sarepta's] stock went down after the AC meeting and went up after FDA sent the June 3, 2016 letter. Dr. Woodcock cautioned that, if Sarepta did not receive accelerated approval for eteplirsen, it would have insufficient funding to continue to study eteplirsen and the other similar drugs in its pipeline.

FDA Commissioner Dr. Robert Califf, Acting Chief Scientist Dr. Luciana Borio, and Dr. Ellis Unger, the Director of the Office of Drug Evaluation, all opposed the approval but Dr. Califf declined to overrule Dr. Woodcock's decision.

Dr. Unger argued that the approval was unethical and counterproductive:

By allowing the marketing of an ineffective drug, essentially a scientifically elegant placebo, thousands of patients and their families would be given false hope in exchange for hardship and risk.

Dr. Borio argues:

Granting accelerated approval here on the basis of the data submitted could make matters worse for patients with no existing meaningful therapies — both by discouraging others from developing effective therapies for DMD and by encouraging other developers to seek approval for serious conditions before they have invested the time and research necessary to establish whether a product is likely to confer clinical benefit.
[...] [Sarepta] has exhibited serious irresponsibility by playing a role in publishing and promoting selective data during the development of this product. Not only was there a misleading published article with respect to the results of Study 201/202147 –which has never been retracted—but Sarepta also issued a press release relying on the misleading article and its findings.

Dr. Derek Lowe, from In The Pipeline, agrees with Dr. Unger and Dr. Borio that the drug is "unlikely to provide much benefit, and is reasonably likely to provide none at all" and that the drug "may well be [$300,000 per year] worth of placebo".

Note: Bold was added by the submitter.

http://endpts.com/senior-fda-officials-warned-that-eteplirsen-ok-would-lower-fda-standards/
http://blogs.sciencemag.org/pipeline/archives/2016/09/20/sarepta-gets-an-approval-unfortunately
https://en.wikipedia.org/wiki/Eteplirsen


Original Submission

Related Stories

In The Pipeline: Coronavirus 45 comments

https://blogs.sciencemag.org/pipeline/archives/2020/01/27/coronavirus

As the world knows, we face an emerging virus threat in the Wuhan coronavirus (2019-nCoV) outbreak. The problem is, right now there are several important things that we don't know about the situation. The mortality rate, the ease of human-human transmission, the rate of mutation of the virus (and how many strains we might be dealing with – all of these need more clarity. Unfortunately, we've already gone past the MERS outbreak in severity (which until now was the most recent new coronavirus to make the jump into humans). If we're fortunate, though, we'll still have something that will be worrisome, but not as bad as (say) the usual flu numbers (many people don't realize that influenza kills tens of thousands of people in the US each year). The worst case, though, is something like 1918, and we really, really don't need that.

[Ed note: The linked story is by Derek Lowe who writes a "commentary on drug discovery and the pharma industry". He is perhaps best known for his "Things I Won't Work With" blog entries which are as hilarious as they are... eye opening. I have found him to be a no-nonsense writer who "tells things as they are", holding no punches. The whole story is worth reading as he clearly explains what a coronavirus is, about the current one that reportedly originated in Wuhan, China, what could be done about it, how long that would likely take, and what can be done for those who have already been infected. --martyb]

Previous Stories Referencing Derek Lowe:

Original Submission

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  • (Score: 3, Insightful) by Fluffeh on Thursday September 22 2016, @12:30AM

    by Fluffeh (954) Subscriber Badge on Thursday September 22 2016, @12:30AM (#404966) Journal

    Wow, this is really scraping the bottom of a barrel here. Allowing a drug like this to go ahead purely because the company needs cash? What happened to the Oath and all that?

    • (Score: 3, Interesting) by bzipitidoo on Thursday September 22 2016, @01:20AM

      by bzipitidoo (4388) Subscriber Badge on Thursday September 22 2016, @01:20AM (#404983) Journal

      If Donald Trump can really shoot someone and not lose any voters, this ever more brazen profiteering is certainly not going to get on the radar.

      This MD drug is just like our current Alzheimer's drugs. They don't cure Alzheimer's. They don't even slow down the progression. All they might do, for some patients, is improve daily functioning, a little.

      Drugs for high blood pressure and high cholesterol actually work in controlling the condition, but, they share the highly profitable property of not actually curing anything. They'd have you believe you have to take the medication for the rest of your life. Doctors might make some noises about changing your diet and getting more exercise, but for many patients the magic pill is easier.

      • (Score: 4, Insightful) by Some call me Tim on Thursday September 22 2016, @03:25AM

        by Some call me Tim (5819) on Thursday September 22 2016, @03:25AM (#405010)

        If Donald Trump can really shoot someone and not lose any voters

        Oh for crying out loud. Hillary could slaughter a school bus full of children on the horns of the Wall Street bull, in broad daylight, and the media would spin it as a right wing anti-abortion protest.
        Answering absurdity with absurdity.

        --
        Questioning science is how you do science!
        • (Score: 4, Informative) by skater on Thursday September 22 2016, @12:50PM

          by skater (4342) on Thursday September 22 2016, @12:50PM (#405103) Journal

          Err...you know he actually said that, right?

          • (Score: 2) by Reziac on Friday September 23 2016, @03:48PM

            by Reziac (2489) on Friday September 23 2016, @03:48PM (#405576) Homepage

            Saying isn't doing.

      • (Score: 2) by Magic Oddball on Thursday September 22 2016, @09:36AM

        by Magic Oddball (3847) on Thursday September 22 2016, @09:36AM (#405069) Journal

        Drugs for high blood pressure and high cholesterol actually work in controlling the condition, but, they share the highly profitable property of not actually curing anything. They'd have you believe you have to take the medication for the rest of your life. Doctors might make some noises about changing your diet and getting more exercise, but for many patients the magic pill is easier.

        High cholesterol is caused by either a mixture of genetic & environmental factors, disorders like hypothyroidism, or medical treatments like dialysis. To quote Wikipedia:
        "Diet has an effect on blood cholesterol, but the size of this effect varies substantially between individuals. Moreover, when dietary cholesterol intake goes down, production (principally by the liver) typically increases, though not always with complete compensation, so that reductions in blood cholesterol can be modest. Genetic abnormalities are in some cases completely responsible for hypercholesterolemia, such as in familial hypercholesterolemia [which] affects about one in five hundred people."

        Regarding high blood pressure, 90-95% of cases involve inherited susceptibility to risk factors (age, weight, salt intake, etc.), but no definitive cause. So different people's cases will be due to different causes, what works great for one person might have no impact on the next, and for the people that don't have any of the 'lifestyle' factors, medication may be necessary to really bring their BP under control.

        (The other 5-10% have hypertension due to a condition like renal insufficiency, and in those cases medication's typically needed unless the underlying disorder can be cured.)

        • (Score: 2) by Reziac on Friday September 23 2016, @04:01PM

          by Reziac (2489) on Friday September 23 2016, @04:01PM (#405584) Homepage

          In fact, hypothyroidism causes high blood pressure two different ways -- by causing calcium to be deposited in arterial walls, and by changing the tone of blood vessels so they're less flexible. I expect its effect on calcium (sucks it out of the blood and deposits it in arteries and joints) also has an effect on potassium balance, which in turn affects BP.

          Also has a direct effect on whether cells can uptake cholesterol (when they can't, more is left in the blood).

          Yet another reason why a complete thyroid workup should be the first thing done, not the last resort.

    • (Score: 4, Insightful) by edIII on Thursday September 22 2016, @01:36AM

      by edIII (791) Subscriber Badge on Thursday September 22 2016, @01:36AM (#404985)

      The medical industry has nothing to do with patient well-being, patient's rights, or ethics. Or even morals.

      I'm not surprised when "more profit" is the sole incentive in this country to do anything. That doctor got paid off, patients will suffer, the disease still rages, and profits keep funneling to a few parasites.

      Standard.Operating.Procedure.

      Just waiting for civil war to start so we can kill all the Pharma execs with guillotines.

      --
      Technically, lunchtime is at any moment. It's just a wave function.
      • (Score: 0) by Anonymous Coward on Thursday September 22 2016, @10:28AM

        by Anonymous Coward on Thursday September 22 2016, @10:28AM (#405075)

        Just waiting for civil war to start so we can kill all the Pharma execs with guillotines.

        You're just a shill of the guillotine industry! :-)

    • (Score: 2) by davester666 on Thursday September 22 2016, @06:09PM

      by davester666 (155) on Thursday September 22 2016, @06:09PM (#405212)

      "I Promise To Do Anything and Everything To Make A Buck For Myself" - oath of a CEO. The "For Myself" part is normally not spoken aloud, just understood by everyone involved.

  • (Score: 2) by black6host on Thursday September 22 2016, @12:34AM

    by black6host (3827) on Thursday September 22 2016, @12:34AM (#404967) Journal

    Once again, figuring out how to get your money for nothing and your chicks for free just ain't that hard! :) (No matter which way this goes.)

    • (Score: 4, Insightful) by bob_super on Thursday September 22 2016, @01:04AM

      by bob_super (1357) on Thursday September 22 2016, @01:04AM (#404976)

      1) look like you're working hard on something critical while you package sugar pills in fancy box.
      2) get the right person to overrule scientists who don't believe in you
      3) sell sugar pills for mind-bogglingly absurd price, per person per year - Use part of the cash to reward 2)
      4) retire to your private island out of the reach of pitchforks.

      • (Score: 2, Informative) by butthurt on Thursday September 22 2016, @02:57AM

        by butthurt (6141) on Thursday September 22 2016, @02:57AM (#405004) Journal

        You seem to be saying that this was a scam from its inception. If that's what you mean, what leads you to that belief? It's not obvious to me that this drug was intended or expected to be ineffective. Several other drugs based on the principle of antisense therapy are being developed:

        As of 2012, some 40 antisense oligonucleotides and siRNAs were in clinical trials, including over 20 in advanced clinical trials (Phase II or III).

        --https://en.wikipedia.org/wiki/Antisense_therapy#Example_antisense_therapies [wikipedia.org]

        According to Sarepta Therapeutics,

        Eteplirsen belongs to a distinct class of novel synthetic antisense RNA therapeutics called Phosphorodiamidate Morpholino Oligomers (PMO), which are a redesign of the natural nucleic acid structure [...] PMOs incorporate modifications to the sugar ring of RNA that protect it from enzymatic degradation by nucleases in order to ensure stability in vivo.

        --http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/PeripheralandCentralNervousSystemDrugsAdvisoryCommittee/UCM481912.pdf [fda.gov]

        It's a complex molecule that contains ribose sugar groups. :-)

        • (Score: 2) by bob_super on Thursday September 22 2016, @04:49PM

          by bob_super (1357) on Thursday September 22 2016, @04:49PM (#405189)

          Not saying that this one started as a scam, just pointing out how easy it would be for a scam to work. You only need enough cash and scientific-looking people to look legit, and the right profit-seeking persons to force it through...
          Not quite unlike Theranos, I guess.

      • (Score: 0) by Anonymous Coward on Thursday September 22 2016, @07:37AM

        by Anonymous Coward on Thursday September 22 2016, @07:37AM (#405056)

        You forgot...

        5) Profit!

        • (Score: 0) by Anonymous Coward on Thursday September 22 2016, @10:31AM

          by Anonymous Coward on Thursday September 22 2016, @10:31AM (#405077)

          I'm pretty sure the "profit" part should go before the "retire" part.

  • (Score: 2, Insightful) by Anonymous Coward on Thursday September 22 2016, @12:35AM

    by Anonymous Coward on Thursday September 22 2016, @12:35AM (#404968)

    That's how big buisiness is conducted these days. "Go down to D.C. and get us a victory! Oh, and take this truckload of money."

  • (Score: 3, Insightful) by Anonymous Coward on Thursday September 22 2016, @01:14AM

    by Anonymous Coward on Thursday September 22 2016, @01:14AM (#404980)

    Hey, this drug is probably worthless, but maybe it's not.... you gonna deny your kid a chance, even if it costs you $300K /yr?

    Thanks, FDA!

    • (Score: 0) by Anonymous Coward on Thursday September 22 2016, @03:09AM

      by Anonymous Coward on Thursday September 22 2016, @03:09AM (#405008)

      Harsh but true: if your kid has DMD, they don't have a chance either way. They will die of suffocation or on a hospital ventilator if they're lucky enough to get that far.

      The correct but unpalatable politically/socially decision is to support them while expending as little resources as possible on minor therapies for people who are already dead. We need an actual generic modification to fix the disease; focus research there. Until there's a cure, though, drugs like this, especially with immense price tags asked to be borne by society, should never be approved for general use.

      • (Score: 2) by sjames on Thursday September 22 2016, @06:47AM

        by sjames (2882) on Thursday September 22 2016, @06:47AM (#405050) Journal

        Best answer, don't approve it, but don't forbid it either. Simply rate it's safety (or lack thereof) for human use with no endorsement for effectiveness and let doctors and their patients decide case by case.

      • (Score: 2) by butthurt on Thursday September 22 2016, @01:13PM

        by butthurt (6141) on Thursday September 22 2016, @01:13PM (#405117) Journal

        I assume you meant to type "genetic modification" not "generic modification." As I understand it, this drug is complementary to messenger RNA (mRNA) that codes for an undesirable protein. The intention was that the drug would combine with the mRNA, inactivating the mRNA before it could reach ribosomes, where the protein would otherwise be made. It isn't genetic modification but it is an attempt to impede the expression of a gene.

  • (Score: 1, Informative) by Anonymous Coward on Thursday September 22 2016, @02:40AM

    by Anonymous Coward on Thursday September 22 2016, @02:40AM (#404998)

    Kratom to be banned in 9 days or so

  • (Score: 2) by Some call me Tim on Thursday September 22 2016, @03:15AM

    by Some call me Tim (5819) on Thursday September 22 2016, @03:15AM (#405009)

    I guess we should start with the lobbyists, then move on to the lawyers.

    /on a happy note, I've got a fan! That's the equivalent to getting a new phone book! I'm somebody now!
    ;-)

    --
    Questioning science is how you do science!
    • (Score: 0) by Anonymous Coward on Thursday September 22 2016, @10:35AM

      by Anonymous Coward on Thursday September 22 2016, @10:35AM (#405078)

      on a happy note, I've got a fan!

      So what? I've got several fans. One on the processor, one on the graphics cars, one on the PSU, …

    • (Score: 2) by bob_super on Thursday September 22 2016, @05:30PM

      by bob_super (1357) on Thursday September 22 2016, @05:30PM (#405199)

      > That's the equivalent to getting a new phone book!

      An annoying useless waste that you wish you could stop?
      Be nicer to your fan, or you won't get a second one.

      • (Score: 2) by Some call me Tim on Tuesday September 27 2016, @10:09PM

        by Some call me Tim (5819) on Tuesday September 27 2016, @10:09PM (#407114)

        Possibly you missed the joke. Remember the Steve Martin movie The Jerk? He's very happy that the new phone book has arrived and says "The new phone books here! I'm somebody now!"

        --
        Questioning science is how you do science!
        • (Score: 3, Touché) by bob_super on Wednesday September 28 2016, @04:12PM

          by bob_super (1357) on Wednesday September 28 2016, @04:12PM (#407476)

          Indeed, I missed it. In my defense, I only saw the movie once, with a friend who (very) loudly laughed just before each joke, making me miss about half of them.
          I should watch it again.

          • (Score: 2) by Some call me Tim on Thursday September 29 2016, @04:10AM

            by Some call me Tim (5819) on Thursday September 29 2016, @04:10AM (#407736)

            No worries, I figured that's what it was. ;-) Watch that one again and then dig up "Better Off Dead" The paper boy is a riot! "I want my two dollars!"

            --
            Questioning science is how you do science!
  • (Score: 2) by RamiK on Thursday September 22 2016, @06:43AM

    by RamiK (1813) on Thursday September 22 2016, @06:43AM (#405048)

    Does it help medicinally? No. But I'm giving hope to thousands of patients and their families and encouraging research efforts and funding by demonstrating the market's demand for the real researches.

    If they take that doctor's license, I'm sure Tobacco, Big Oil or Firearms will snatch that guy first chance they get.

    --
    compiling...
  • (Score: 3, Insightful) by FatPhil on Thursday September 22 2016, @06:51AM

    by FatPhil (863) <{pc-soylent} {at} {asdf.fi}> on Thursday September 22 2016, @06:51AM (#405052) Homepage
    The "Center for Drug Evaluation and Research", I presume has the remit of Researching, and Evaluating, Drugs.

    The "FDA", apparently Administers approvals of Drugs.

    But the FDA had a "review team" who were performing an "analysis" - that's researching and evaluating, isn't it? That's CDER's job, according to their name.

    However, the CDER "approved" this drug, despite that being the FDA's job.

    !?!??! Cock goes where?
    --
    Great minds discuss ideas; average minds discuss events; small minds discuss people; the smallest discuss themselves
    • (Score: 1, Informative) by Anonymous Coward on Thursday September 22 2016, @07:48AM

      by Anonymous Coward on Thursday September 22 2016, @07:48AM (#405058)

      All of CDER is FDA, but not all of FDA is CDER.

      Hope that helps.

  • (Score: 0) by Anonymous Coward on Thursday September 22 2016, @08:56PM

    by Anonymous Coward on Thursday September 22 2016, @08:56PM (#405278)

    A friend told me about SRPT a while ago and I put some money into it over the years. While I'm glad the stock price has popped to a multiple of what I paid, my non-expert self does wonder whether the drug is actually efficacious.

    • (Score: 0) by Anonymous Coward on Thursday September 22 2016, @09:21PM

      by Anonymous Coward on Thursday September 22 2016, @09:21PM (#405287)

      Their trials do not show convincing evidence of efficacy and their surrogate endpoint is shit. You can find a link to the FDA review summary from the second link.

  • (Score: 2) by butthurt on Thursday September 22 2016, @09:46PM

    by butthurt (6141) on Thursday September 22 2016, @09:46PM (#405294) Journal

    My understanding of this is scanty, but I see a possible way that it could be ineffective. As I understand, the drug is an RNA molecule that's been modified so it won't be metabolised. It binds to messenger RNA that codes for an unwanted protein. So when a cell makes that messenger RNA, the drug binds to it, forming a combination that, I assume, resists decomposition by the cell. That would use up the drug. Whatever regulates the expression of genes may sense that the mRNA or the protein aren't present. Since the cell's DNA remains unchanged, that DNA can direct the production of additional messenger RNA. The amount of the drug that's administered is limited, but the "ingredients" for RNA are abundant so the cell may be able to produce more mRNA molecules than there are available Eteplirsen molecules (stoichiometry), and dystrophin will be made.

    • (Score: 0) by Anonymous Coward on Friday September 23 2016, @12:50PM

      by Anonymous Coward on Friday September 23 2016, @12:50PM (#405506)

      You're probably right. The idea should work, which is why it was called a "scientifically elegant placebo", but the levels of mRNA restored with the frequent injections are still too low for reliable detection.