Urge the FDA to Say YES to Accelerated Approval for safe, effective therapies for children with Duchenne.
We urge the Food and Drug Administration (FDA) to use the Accelerated Approval pathway for approval and access to safe, effective therapies for Duchenne Muscular Dystrophy - the leading genetic killer of children that impacts 1 out of every 3,500 boys born in the United States. It's time for the FDA to Say Yes and make this the first generation of Duchenne survivors.
Created: Feb 27, 2014
Issues: Disabilities, Health Care
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The only thing these bureaucrats are really adept at is frankly being a bureaucrat. The FDA has an annual budget north of $4.5 BILLION, and while they do a lot of good, their inconsistency in granting approval leaves me questioning their impartiality.
We need a replay booth like the NFL to get the call right. And in this case, it is a touchdown.
This really should be one of the easiest decisions the FDA ever gets the opportunity to make. No real alternative treatment, no efficacy issues, over two years of data, multiple videos of DMD boys getting to act like boys, attendance by FDA officials at a recent 6MWT, and so forth. There shouldn't be any real pushback from outside groups, but alas, we do not live in a perfect world. How could anyone be "uncomfortable" ruling favorably?
It is sickening decision makers can be "flippant" to some of the greatest DMD minds in the world and get away with it, all the while dragging out a ruling in the name of public safety. Those specialists have literally 100's of years studying this disease.
I'm surprised Christine, Jenn and the other vocal parents have any sanity left.
Absolutely amazing awesome alliteration.
And your possible explanation does make sense. I'm old school - somebody buys means somebody sells. I wish I really understood how the market makers (manipulators) really did it.
I've been long in this stock since before some of these kids were even born, so my sentiments (and $$$) lie on the side of the boys and the company. This bureaucracy, while governed by law, operates as an autonomous and almost rogue agency that seemingly answers to no higher authority. And we saw last November what can happen even when it appears all is in order. We all want to believe common sense will prevail, but as Yogi Berra famously said, "it ain't over till it's over".
You're sitting at home one day and you realize your house is on fire. So you pick up the phone.
"Hello, Fire Dept of America (FDA), J. Woodcock speaking on a recorded line, how may I help you?"
"Uh, yes my house is on fire and I need you guys to come over and put out the fire."
"Well, yes, we'd love to, but can you assure us the house will actually burn down if we don't respond?"
"Yes, I'm positive it will if you don't come over right now!"
"Okay, please don't get upset with me. Let me run it past our crack team here. I'm sure we will be able to get something done, but even though I can make a decision, I will need to make sure the entire team is on board with the decision to assist you. In the meantime, could you please forward information on what materials were used to construct the house, how old the house is, any comparative houses like yours in the neighborhood that experienced similar fires, and oh, throw in some information on houses that haven't burned so we may be able to make the correct decision. And as soon as you get the info assembled we will call you in to review the paperwork and make sure everything is in proper order. And thanks for calling the Fire Dept of America. Have a nice day."
We are all frustrated by the performance of the stock, but rather than aim at the stock and company management, refocus on:
1. A government bureaucracy that seemingly won't follow the law;
2. Competing companies who know no ethics and think nothing of manipulating or withholding data;
3. So-called on-line publications that allow biased "experts" to pound the stock if it starts showing life;
4. An unwillingness (at least up to now) of that government bureaucracy to ignore the call of parents and experts from across the globe calling for quick approval.
I am sure there are a host of other solid reasons I have overlooked, but you get my point.
I have two boys, both of whom are grown and healthy. When they were both born it was a total relief to have the medical staff say everything was good.
I know the FDA is under much scrutiny and pressure from many groups. And I know being methodical can quite often be the right approach. In the case of DMD, though, we are up against a relentless and certain killer.
Here we have parents willing to fight as hard as they possibly can for the survival of their boys. They have traveled the country telling their story, appeared before Congress, allowed their boys to be turned into human guinea pigs, and all in the name of science. These boys are true pioneers.
We also have a government that, under certain circumstances, will arrest parents for withholding medical treatment to their children and take away their children.
I don't see how we as a country can have it both ways. There can be no harm in, at the very least, a conditional approval, with a Phase 3 to run concurrent with the conditional approval.
And I think we can all agree the boys on this drug can and will be monitored for the rest of their lives.
And 11 minutes later I got a response from Dr. Woodcock:
Thank you for writing. We will take your comments and position into account. Janet Woodcock
What's dumbfounding is there are other governmental agencies that will take kids away from parents who DON'T provide proper medical care based on religious or other beliefs. The hypocrisy of it all.
I think you are correct about the reporting date. What I saw for Form 13-F (which I think is the form used) is 45 days after the end of the quarter. Next couple of weeks could get real interesting.
Couple all this with RNA's CEO coming out this week saying maybe things aren't what they seem with Prosensa after GSK abandoned RNA, LeeRink upgrading RNA, and we have the ingredients for one of the most compelling soap operas of all time.
I'm guessing the FDA has their blender, washing machine, tops, hula hoops, anything that spins, looking for the "approval answer".