New Drug For Rare Childhood Disease – Price Tag: $702,000 Per Year!…AND An EXPLOSIVE Statement From The FDA – By Jon Rappoport – May 1, 2017

Posted: May 1, 2017 in Uncategorized

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…”

From Rex – The Press Release On This:

FDA Approves World’s Most Expensive Drug to Include UCD Children as Young as 2 Months [Link: http://www.raredr.com/news/fda-ucd-2-months]

By James Radke Published Online: May 01, 2017

The FDA has lowered the age at which children with urea cycle disorders (UCDs) can take Ravicti (glycerol phenylbutyrate). 

Now, patients as young as 2 months of age are approved to ingest the oral liquid medicine.

Ravicti, while safe and effective, is probably best known as being the most expensive drug in the world with an annual cost of $793,632. 

In 2013, the drug was originally approved for UCD patients 2 years of age and older.

The age expansion was approved based on 3 studies assessing monthly ammonia control and hyperammonemic crises in UCD patients aged 2 months to 2 years.

Data from 1 of the studies was presented at the ACMG Annual Meeting in Phoenix earlier this year. 

Berry reported on 17 UCD patients aged 2 months to 2 years of age who were switched from sodium phenylbutyrate to equivalent doses of glycerol phenylbutyrate.

In that study, 82% and 53% of patients completed 3 and 6 months of therapy, and patients maintained excellent control of ammonia and glutamine levels (Table 1).  

Prior to enrollment, 11 patients had 36 hyperammonemic crises, and after switching to glycerol phenylbutyrate, 6 patients had 10 hyperammonemic crises with a reduction in their annual rate (from 2.98 to 0.839 episodes).

Four (23.5%) patients reported 1 non-serious adverse event considered drug-related: 2 skin problems, 1 diarrhea, and 1 elevated ammonia.

Table 1.  Glutamine and Normalized Plasma Ammonia Levels over Time
Visit – Normalized Ammonia(µmol/L) – Glutamine – (µmol/L)

Baseline – 89.16 ± 63.1 (n=17) – 677.1 ± 244.9 (n=13)
Month 3 – 62.24 ± 70.3 (n=13) – 529.4 ± 123.6 (n=10)
Month 6 – 40.32 ± 23.4 (n=8) – 609.4 ± 210.8 (n=7)
Month 12 – 30.88 ± 23.7 (n=6) – 505.7 ± 310.9 (n=6)

In a news release (http://www.globenewswire.com/news-release/2017/05/01/974919/0/en/Horizon-Pharma-plc-Announces-FDA-Approval-to-Expand-the-Age-Range-for-RAVICTI-glycerol-phenylbutyrate-Oral-Liquid-to-People-with-Urea-Cycle-Disorders-Two-Months-of-Age-and-Older.html), Susan Berry, M.D., professor and division director for genetics and metabolism, department of pediatrics, University of Minnesota said, “This approval represents a significant advance for very young children with UCDs, one of the most vulnerable patient populations, as more severe cases of the disease tend to present earlier in life and can lead to serious long-term impairments if not diagnosed and treated early.

”Urea cycle disorders (UCDs) are genetic disorders that results in a deficiency of one of the enzymes in the urea cycle.

The onset and severity of urea cycle disorders is highly variable and can occur in either children or adults. 

Severe forms of the condition are often caught early in life with symptoms such as: seizures, hypotonia, respiratory distress, and coma occurring. In other cases, children may go asymptomatic for several years. 

A common misdiagnosis in these children is Reye’s syndrome.

Adults with urea cycle disorders may display symptoms such as of disorientation, confusion, slurred speech, unusual and extreme combativeness or agitation, stroke-like symptoms, lethargy and delirium. Without proper diagnosis and treatment, these individuals are at risk for permanent brain damage, coma, and death.

Reference

Berry S Longo M, Robinson B, Vockley J. Safety and Efficacy of Glycerol Phenylbutyrate for Management of Urea Cycle Disorders in Patients Aged 2 Months to 2 Years. Presented at the ACMG Annual Clinical Genetics Meeting; Phoenix, AZ; March 21-25, 2017.

https://jonrappoport.wordpress.com/2017/05/01/new-drug-for-rare-childhood-disease-price-tag-702000-a-year/

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