The Food and Drug Administration on Thursday approved BioMarin Pharmaceutical's drug Brineura as the first treatment for a specific form of Batten disease, an extremely rare nervous system disorder.
Symptoms of the disease — including language delay, recurrent seizures and poor movement coordination — appear in affected children starting at age 2 to 4.
Brineura, an enzyme replacement therapy, slows down the disease's progression and allows children to maintain walking ability longer. The drug is administered into the patient's cerebrospinal fluid via catheter every other week.
The drug will cost $27,000 per biweekly infusion, or about $702,000 a year, reports Bloomberg. BioMarin on Thursday said most Batten disease patients rely on federal assistance programs to help pay for treatment. The drug's cost after mandatory government discounts will be $486,000, according to BioMarin.
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