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Cystic Fibrosis Gene Correction Drug Approved by the FDA 264

tguyton writes "The good news: the FDA just approved the distribution of the first drug to treat the underlying cause of Cystic Fibrosis, called Kalydeco by Vertex Pharmaceuticals. The bad news: this drug will only affect 4% of patients with the disease in the U.S. From the article: '[Affected patients] with the so-called G551D mutation have a defective protein that fails to balance the flow of chloride and water across the cell wall, leading to the buildup of internal mucus. The vast majority of cystic fibrosis patients have a different genetic defect, in which the protein does not reach the cell wall. Vertex is developing another drug to try and address that problem. Study data for that drug is expected later this year.' Hopefully the research involved will be applicable to finding treatments for other genetic diseases." Further bad news: "...executives said Kalydeco would cost $294,000 for a year's supply, placing it among the most expensive prescription drugs sold in the U.S."
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Cystic Fibrosis Gene Correction Drug Approved by the FDA

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  • What is the co-pay on that? :)
    • The good news is you will meet your "out of pocket cap" in a few hours.

      The bad news is your insurance won't approve of the drug until the generics come out... some time in 2064.

    • by rwade ( 131726 ) on Wednesday February 01, 2012 @01:12PM (#38892771)

      From my understanding, those suffering from CF in California generally have their bills picked up by the state since no insurance plan could ever afford to treat CF patients. I believe this is under the state's Genetically Handicapped Persons Program [].

      But not having CF myself, I'm certainly no expert on the cost of care for it...

      • Which States are bankrupt and why?
        • by jeko ( 179919 ) on Wednesday February 01, 2012 @02:58PM (#38894303)

          I could point out that California isn't bankrupt because of healthcare costs. I could do a whole excrutiatingly exhaustive review of just why California's revenues got strangled. I won't, because it won't matter to you.

          Since you're doing your thinking emotionally -- the shrinks nickame it the "Just World Hypothesis" -- let me see if I can counterbalance the fear you're suffering from by adding a different one.

          If a civilization has any obligations -- any at all -- then caring for the vulerable is the first among them. Any system that can't -- or won't -- care for the very young, the very old and the physically infirm doesn't deserve to continue. I know Sparta sounds cool, but I'm kinda glad a people who threw babies against rocks and committed murder for sport aren't around any more. In fact, I would argue that their brutality is precisely WHY they're not around any more. People who are working together tend to weather crises far better than a group of jackasses standing around screaming "Only the Strong Survive!" Teamwork, you know? Maybe your coach mentioned it? Remember John Wayne screaming back, "That's WHAT I got?" No? OK. didn't think so.

          I'm old enough now to have watched a few of my friends and some of my family die. Heart disease is bad. Diabetes is worse. Cancer is flat-out evil. Real "We had to pick up a knife to save you" surgery is damn near the same thing as surviving a stabbing. I know, I know, those are just words to you. Let me put it in terms you may understand. You'd much rather be eaten by a vampire than succumb to cancer. You'd prefer any videgame death to what most fatal diseases have in store for you.

          Here's where I'm going to pull back my hood and tap you on the shoulder with a long, bony finger. Eat a perfect diet. Exercise all you want. Revel in whatever gifts youth and good health can provide. I'll still be waiting. I got Steve Jobs. I got Feynman. I got Newton. You think I'm going to miss you? At 35, you'll notice you've lost a step. At 45, you and your doctor will have a little chat. At 55, those chats become discussions. At 65, you begin long talks about the options you have remaining.

          Believe me when I say they'll dwindle.

          And this is the best possible outcome, assuming some little patch of slippery ice has't gotten you first.

          So while you're sitting there blithely saying we should kick the sick and the weak to the curb, I'm smiling. Because I know you'll be among them soon enough.

          And people like you always whine the loudest when I come.

  • Cell MEMBRANE (Score:5, Informative)

    by Anonymous Coward on Wednesday February 01, 2012 @12:50PM (#38892477)

    Good lord, we are animals not plants. There is no such thing as a "cell wall" in our cells! Call it what it is: the cell membrane.

    Pedantic? Yes, but the definitions are precise and are intended to be used precisely. Journalism like this makes me want to gouge my eyes out; a single high-school biology class teaches cell wall vs. cell membrane!

    • by robotkid ( 681905 ) <> on Wednesday February 01, 2012 @02:58PM (#38894297)

      Good lord, we are animals not plants. There is no such thing as a "cell wall" in our cells! Call it what it is: the cell membrane.

      Pedantic? Yes, but the definitions are precise and are intended to be used precisely. Journalism like this makes me want to gouge my eyes out; a single high-school biology class teaches cell wall vs. cell membrane!

      I'm a scientist., I'll handle this!

      By the power vested in me by science, I hereby retroactively flunk the original submitter's high school biology grades, and also raise the grade of the bookish, socially awkward lab partner you conned into doing all your work. The sentence is to correct 10 obnoxiously factually incorrect slashdot comments without invoking any of the following: Godwin's Law, correlation vs. causation, Ron Paul, or conspiracy theories of any kind. Oh, and just for good measure, rule 34.

      Until then your slashdot submitting privileges are subject to double-secret probation.

  • by vlm ( 69642 ) on Wednesday February 01, 2012 @12:51PM (#38892491)

    Just so no one gets confused, this molecule goes by 3 common names VX-770 and Ivacaftor and Kalydeco []

    There are not three separate drugs for the same problem etc.

    • by the gnat ( 153162 ) on Wednesday February 01, 2012 @01:36PM (#38893155)

      Just so no one gets confused, this molecule goes by 3 common names VX-770 and Ivacaftor and Kalydeco

      I asked a scientist I know at a Big Pharma company why there are always multiple names. His response was (paraphrased): "There's an entire department at our company full of people who come up with names for drug candidates - one name that's recognizable and easily pronounced which becomes the trademarked brand name, and another that's hard to pronounce or remember that becomes the official compound name, which is what the generics will eventually use." (I think he was exaggerating about it being an entire department, but I could be wrong.)

      If you pay any attention to biomedical literature, they always use the compound name - thus "Prozac" will always be referred to as "fluoxetine", "Gleevec" is "imatinib", and so on.

  • Can anyone explain a bit about how this drug works? I understand CF is caused be a genetic "error", but is this an exon skipping drug (similar to what they're working on with muscular dystrophy) or is this something different?

    • by PenguinX ( 18932 )

      According to NPR, Kalydeco, "works by helping to fix one defect in the protein that causes the disease." Unfortunately the way the drug works is also very specific, and won't work for all sufferers NPR also reports that it will "only work for about 1,200 patients in the U.S.". Now, being that a cursory Internet search says that there are about 30,000 sufferers in the USA, it's pretty clear that Kalydeco is just a step in the right direction, at least from a medical and research perspective.

      http://www.npr.or []

    • Re:Exon Skipping? (Score:5, Informative)

      by Cyclizine ( 2558090 ) on Wednesday February 01, 2012 @01:21PM (#38892923)
      CF is caused by defects in the CFTR protein, an ion channel on the surface of many cells, including the cells that line the respiratory tract. Basically, it creates an osmotic potential by moving chloride and other ions from the cell to the outside, so water flows out of the cell into the mucous in the airways, making it less viscous. I'm an anaesthetist, not a respiratory physician, but as far as I understand, in the F508 mutation (most common ~70%), CFTR doesn't even make it onto the cell membrane. In the G551D mutation, CFTR reaches the cell membrane, but degrades more rapidly than normal. Ivacaftor acts to increase the length of time the faulty protein stays on the surface until it's degraded. Hence why it's of no benefit in the most common mutation.
  • Further bad news: "...executives said Kalydeco would cost $294,000 for a year's supply, placing it among the most expensive prescription drugs sold in the U.S."

    Early adopters can use it, and the price will be driven down for everyone, just like the cost of sequencing your own genome.

    My question is, what drug is more expensive?

    • by vlm ( 69642 )

      My question is, what drug is more expensive?

      Idursulfase for the win. Interesting from a biochemical production standpoint. []

      You can get into definition battles, whats more expensive to society, a quarter million for one dude who actually needs it, or 100K unneeded $10 lorazepam prescriptions for people who don't really need it ...

      • by tmosley ( 996283 )
        "Need" doesn't justify the collective burdening of others. Sure, "we" could easily absorb the cost of one of these types of things, but what happens when there are thousands, or millions of similar cases? Who gets to decide who gets access to everyone's money? It is a system that breeds corruption, and will cause eventual collapse, and greater harm to most of the participants.

        As cruel as it sounds, it is the market that must decide. It is the only way to keep things anywhere close to fair for EVERY
        • Just be careful that the "market" doesn't turn out to be a whole lot of people tired of being poor, sick and with no prospects of moving up taking out their rage on the rich and healthy people who keep talking about how the market will fix everything.

          You people ought to investigate why we have a social safety net. Only half of the reason is to protect the poor and the sick. The other half is to protect the rich from the poor getting their pitchforks out.

          • by tmosley ( 996283 )
            But we need them to get their pitchforks out. The system is corrupt to the core.

            I will not be party to the provision of the mob with bread and circuses to protect cruel and incompetent leaders.
            • So you'd rather live in the times of strife where the poor were abjectly poor (and rather permanently poor), where the rich feared the poor at every turn, and where revolutions were fairly regular? You should put down your rose-colored glasses. Those times sucked for everyone.

        • It might sound better to just say that health care policy is best not driven by fringe cases like this. You base policy out to, say, 2 or 3 sigmas, and handle anything beyond that on a case by case basis.

          Would help to have an efficient government where office holders consult actual experts on complex topics, though, and that's where the whole idea sort of falls about, you see. :-\

          • by raydobbs ( 99133 )

   mean...someone -BESIDES- accountants and business analysts, who make absolute decisions based on stock option growth and corporate profitability?

        • by fedos ( 150319 )
          And, as usual with "the market will fix everything" crowd, "everyone" is a code word for "the rich", i.e. "a chosen few".
    • by rgbrenner ( 317308 ) on Wednesday February 01, 2012 @01:02PM (#38892657) []

      Alexion Pharmaceutical's Soliris, at $409,500 a year, is the world's single most expensive drug. This monoclonal antibody drug treats a rare disorder in which the immune system destroys red blood cells at night. The disorder, paroxysymal nocturnal hemoglobinuria (PNH), hits 8,000 Americans.

      Elaprase ($375,000 per year) treats an ultra-rare metabolic disorder called Hunter's syndrome. Just 500 Americans suffer from the disease, which causes infections, breathing problems and brain damage.

      Naglazyme from BioMarin Pharmaceuticals treats another rare metabolic disorder and costs $365,000 a year, according to investment bank Robert W. Baird. Viropharma predicts that sales of its Cinryze, a treatment to prevent a dangerous swelling of the face, will increase from $95 million last year to $350 million several years from now. The drug costs an estimated $350,000 a year.

      • Interesting. It seems the most expensive drugs are expensive because they treat diseases that are rare, thus they don't get the advantage of spreading the research cost out over many sales.
      • it really is; glad they're around, but you ever notice they never have a cure? Not even if it was half a mil for a one-time cure; their business is repeat customers with literally life-long lock-in.

        Big Pharma: drug dealers in every sense of the phrase.
  • Obviously they're playing chicken with the insurance companies, trying to get them to pay out the nose. But would it be better to not publicize these expensive innovations, but only make them available to rich doctors that treat rich patients? Then after the R&D has been recouped, release the knowledge of them to patients of more limited means.

    Otherwise, you're just dangling it in front of the poor/ uninsured. Hey, we could keep your kid alive, but neener, neener, neener.
    • Otherwise, you're just dangling it in front of the poor/ uninsured. Hey, we could keep your kid alive, but neener, neener, neener.

      Isn't that how a for-profit medical system works?
      • Sure, but the US doesn't have one of those. There are laws that a hospital/doctor has to treat the patient in front of them. (If it's life or limb threatening)
        • by sjames ( 1099 )

          So they keep the kid alive then hound you through every legal means (and a few illegal ones thanks to debt collector ethics) for the rest of your (now) miserable existence,. Great.

    • by raddan ( 519638 ) *
      Actually, Vertex is offering the drug free of charge to anyone who is uninsured and who makes less than $150,000/yr.

      Given the cost of development, and the small merket, this sounds pretty reasonable to me.
  • Worth It (Score:3, Interesting)

    by Anonymous Coward on Wednesday February 01, 2012 @01:26PM (#38892995)

    Posting anon 'cause I've already modded.

    My wife has MS and takes Tysabri (Natalizumab).
    It started off costing us (and the insurance co.) $6000 a month. However Bio-gen began a copay/deductible assistance program after they saw how the price was keeping people from access to the drug. This has made our costs manageable. And I'm sure it's helped hundreds, if not thousands of people.
    I bet (and hope) the same thing will happen to this drug.

  • I would think 294k would make it the MOST expensive drug... I have to imagine there is some chemotherapy that are expensive... but that seems insane... considering that it doesn't cure the problem, just mitigates it.
  • Saddly it's about a decade too late for my college roomie with CF. He was brilliant and very funny, a good friend and a great programer. We're all a bit poorer every day this disease continues to kill.

  • by medv4380 ( 1604309 ) on Wednesday February 01, 2012 @01:55PM (#38893477)
    The best way to deal with Cystic Fibrosis is for Carriers to go the IVF route so they don't pass the gene on to their kids. All this "CURE" will do is make it so that CF isn't as lethal making it propagate out as a benign disease when in truth it will just be making a population dependent on drugs to live. A real Cure would be a gene therapy that removes the defect from every cell in the patients body so they don't have to continue taking it for the rest of their life, and have no risk of passing it on to their kids.
    • Have you not been paying attention to how the American medical industry works these days? Why cure it once, when you can treat it indefinitely? There's no benefit to the shareholders by catering to short term customers.
      • Yep, an ounce of prevention or treatment is worth a pound of cure. When stretched out over the lifetime of a patient.

  • by ABadDog ( 28370 ) on Wednesday February 01, 2012 @02:21PM (#38893835) Homepage

    Vertex has a very generous patient assistance program for this drug. It's *free* for those without insurance and who make less than $150k/year, and for others there's a generous co-pay assistance program. This drug will be available for everyone who needs it. We've spent 10 years and I don't even know how many hundreds of millions of dollars developing this drug, and helping save lives is what keeps us motivated every day.

    of course, I don't speak for Vertex.

  • I've got karma to burn so I'm going to ask this here.
    The summary refers to the company planning on "developing another drug to try and address that problem." While I understand that most language, and particularly English, is ultimately defined by usage rather than by formal standards, as someone for whom English is not his first language, I find myself flabbergasted by the "try and" idiom. I can understand "try to" as it makes logical sense; I don't see, however, the logic behind "try and" as it implies t

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