New drug for rare childhood disease; price tag, $702,000 a year
And an explosive statement from the FDA
by Jon Rappoport
The FDA has just approved a new drug, Brineura, for a rare
childhood brain-disease known as CLN2, which progresses quickly and is
considered fatal.
BioMarin, the drug's manufacturer, has pegged the yearly
price of the drug for a patient at an astronomical $702,000. Discounts
will be offered---the average annual price for the patient will come
down to $486,000.
While recovering from the pricing shock, consider this
statement from the FDA: "Brineura is the first FDA-approved treatment to
slow loss of walking ability (ambulation) in symptomatic pediatric
patients 3 years of age and older..."
Translation: There is no claim that the drug cures the
disease. The drug may slow down the progression of only one of the many
symptoms. For $486,000 a year, if the patient lives for a year.
Well, we'll see whether even that "slowing down" assertion
pans out, because the clinical trial of the drug was carried out on only
22 children, to determine efficacy, and 24 children to determine
safety.
Of course, the drug's manufacturer would state that, since
CLN2 is a very rare disease, it wasn't easy to find patients on whom to
test it. Nevertheless, the FDA approval for the drug was based on scanty
evidence---to say the least.
It's fairly clear that researchers and drug companies look at
this situation as a first step in developing more (highly expensive)
drugs to treat CLN2. The gateway has been opened. Though they wouldn't
admit it, Brineura is an experimental medicine, and if follow-up doesn't
record a high percentage of deaths occurring sooner than expected
(according to what parameters?), it will be considered a great success.
Here is how the drug is invasively administered. Keep in mind
that the patient is a very young child who is already unable to
function in the world, is confused, is having great difficulty walking
and even sitting:
FDA press release (4/27/17): "Brineura is administered into
the cerebrospinal fluid (CSF) by infusion via a specific surgically
implanted reservoir and catheter in the head (intraventricular access
device). Brineura must be administered under sterile conditions to
reduce the risk of infections, and treatment should be managed by a
health care professional knowledgeable in intraventricular
administration. The recommended dose of Brineura in pediatric patients 3
years of age and older is 300 mg administered once every other week by
intraventricular infusion, followed by an infusion of electrolytes. The
complete Brineura infusion, including the required infusion of
intraventricular electrolytes, lasts approximately 4.5 hours.
Pre-treatment of patients with antihistamines with or without
antipyretics (drugs for prevention or treatment of fever) or
corticosteroids is recommended 30 to 60 minutes prior to the start of
the infusion."
Shocker: I found this explosive statement in FDA press
release: "The initial symptoms [of the childhood disease CLN2] usually
include language delay, recurrent seizures (epilepsy) and difficulty
coordinating movements (ataxia). Affected children also develop muscle
twitches (myoclonus) and vision loss. CLN2 disease affects essential
motor skills, such as sitting and walking. Individuals with this
condition often require the use of a wheelchair by late childhood and
typically do not survive past their teens."
Does that sound like anything you've ever heard of?
It's the result, in some children, of administered vaccines.
Vaccine damage.
Imagine this. A doctor says to a mother: "You have to stop
talking about the horrible things that happened to your child right
after he received a vaccine. You're wrong. You're not making sense. It
wasn't the vaccine. Your child has a rare genetic brain disorder called
CLN2. We now have a drug that may slow down one of the progressing
symptoms. It'll cost $486,000 a year. To give the drug, we'll need your
child for five hours every other week. We'll insert a catheter in his
head..."
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