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.
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…”