Apr 27, 2017

New drug for rare pediatric disease will cost $702k per year

David Duprey / AP

The FDA approved Brineura on Thursday, the first drug to treat a rare, fatal nervous system disorder called Batten disease that affects children. BioMarin Pharmaceutical makes the so-called orphan drug and will charge $27,000 for treatment every two weeks, or $702,000 per year, executives said Thursday. That does not include discounts from Medicaid or other discount programs.

That's a huge price tag, but that's not all: BioMarin secured another priority review voucher, which allows a company to get a speedier drug review from the FDA. BioMarin sold its first voucher in 2014 for $67.5 million.

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