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FDA just approved one of the most expensive drugs in the world

By Karen Graham     Apr 29, 2017 in Health
On Thursday, the U.S. Food and Drug Administration (FDA) gave its approval for the drug, Brineura, manufactured by BioMarin Pharmaceutical Inc. Brineura also comes with a list price of more than $700,000 a year.
While the price of Brineura may seem outrageous to most people, it is the first ever drug to be manufactured to treat a rare form of Batten disease, a fatal, inherited disorder of the nervous system that typically begins in childhood.
The drug will be used in patients with late infantile neuronal ceroid lipofuscinosis type 2 CLN2, a variant of Batten Disease in which there is a deficiency of an acid protease, an enzyme that hydrolyzes proteins.
BioMarin estimates that between 1,200 to 1,600 children across the globe have the disease with 85 percent living outside the U.S. These children suffer with multiple symptoms, from seizures to trouble coordinating muscles to vision loss and eventually become bedridden. The medicine is approved for children at least three years old.
And while BioMarin should be commended for making a treatment available for such a rare disease, many people are shocked by the high price of the medication. And to many people, this is just another example of "price-gouging" by the pharmaceutical industry.
However, patients will not pay the full amount due to discounts, rebates, and patient assistance programs. BioMarin, like other drug companies who focus on rare, or "orphan" diseases and disorders, justifies the high prices, arguing that it's difficult to make breakthroughs on drugs for diseases affecting a small percentage of the population.
New drugs go through clinical trials, and for rare diseases, there is a limited pool of subjects that can be used. There is also a very small pool of patients that would even be interested in the drug, making marketing difficult. This actually happened with Glybera, a $1.0 million-plus gene therapy from UniQure for a rare metabolic disorder. After five years with hardly any sales in Europe, the product was shelved.
But Brineura isn't the most expensive medicine on the market today. There is Horizon Pharma's Ravicti, retailing for a cool $793,632 per year. Ravicti is a nitrogen-binding agent for chronic management of adult and pediatric patients at least two years of age with urea cycle disorders (UCDs). In UCDs, nitrogen accumulates in the body in the form of ammonia, a highly toxic substance that can cause irreversible brain damage, coma, and even death.
More about grugs, Fda, Batten's disease, Controversy, Drug prices
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