Exclusivity: How The Government Keeps Orphan Drugs in Production

April 22, 2014

Why did the FDA grant BioMarin this permission, known in bureaucratic lingo as a “six-month pediatric exclusivity extension?”

BioMarin Pharmaceutical in Novato and San Rafael has six more months to keep exclusively selling a new, kid-friendly form of its drug for a disease called phenylketonuria.

Haven’t heard of phenylketonuria? That’s because it’s a rare genetic disorder that only affects 10,000 to 15,000 people in the United States, which makes it a so-called “orphan” disease (in the U.S, these are disorders that affect fewer than 200,000 people).

Patients with phenylketonuria can’t process an amino acid called phenylalanine found in many foods, so it ends up building up in the blood. Left untreated, high blood phenylalnine levels can affect the brain, impair thinking and cause behavioral problems.

BioMarin’s treatment for the disease is called Kuvan, and it’s been on the market since it was approved in 2007. It costs an average of $90,000 a year, and last year it made $167.4 million. And this week, the U.S. Food and Drug Administration said it will allow BioMarin to keep selling the drug, without competition, until June 2015. The drug has been available as a tablet, but now it is also a powder that’s intended for infants and small children: it can be dissolved in water, apple juice or soft foods.

The reason BioMarin is in the business of rare diseases in the first place goes back to 1983, when the nation passed the Orphan Drug Act. Before then, few companies made drugs for orphan diseases because there were relatively few patients who would buy the treatments. To motivate companies to make drugs for these diseases, the law gave such companies the right to sell the therapy for seven years without competition.

Another problem: Pharmaceutical companies have historically been reluctant to do clinical trials on children. The market for children patients is small (compared to adults), and it’s hard to get informed consent (kids may not understand what’s going on during a trial).

Read more at original source.

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