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."
Worth It (Score:3, Interesting)
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.
Re:Same thing as VX-770 and Ivacaftor (Score:4, Interesting)
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.
Not even Laffer agrees with that any more... (Score:5, Interesting)
Laffer has been proven correct thus far
Yeah, except that not even Laffer agrees with that any more. He's been backpedaling furiously from a bad theory made 30 years ago that's been empirically, wondrously disproven over the past ten. Abondoning his little napkin sketch was the only way he could retain a shred of academic credibility.
You simply cannot provide everyone with all the healthcare they want
Sure, sure, sure, just answer me this.
Why do we give federal subsidies to Harvard Medical School?
Because they threaten to train more doctors if we don't. We grant them a federal subsidy to restrict admission because the American Medical Association says that too many doctors in the field will lead to a lower standard of living for doctors.
And it's not just Harvard. Every medical school is granted subsidies to restrict enrollment.
Hmm. Seems odd, doesn't it? We can't find enough resources to meet America's medical needs in much the same way that companies can't find enough American engineers to fill all the jobs.
But let's assume those nonsense numbers are true. Let's try this. How about we divert all of the resources from the current War on Drugs and War on Terrorism and redirect those trillions of dollars to a War on Illness? Surely we can agree that a few more Harvard-educated medical doctors would do more good then a few thousand more TSA agents.
How about we find all the kids bright enough to become doctors and sponsor them through medical school? How about we devote research dollars to more than just making sure rich guys can screw their trophy wives?
How about we agree with all the drugs companies that government is wasteful and inefficient, and that we welcome their competition when we start opening drug factories they same way we open utility companies. How about when they start whining about horrendous research costs, we tell them we couldn't agree more, which is why we're going to ask them to pay back all the money we gifted to them over the past six decades.
How about we take Manhattan and Apollo project resources for the next 20 years and apply them to healthcare? Then let's see if your nonsense about "We can't afford to take care of our own" falls apart into the same pile of bull that the Laffer Curve and Supply-Side economics did.