FDA approves Brineura for Batten disease

FDA approved a BLA from BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) for Brineura cerliponase alfa (BMN 190) to treat children with neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a form of Batten disease. The therapy is the first approved for the indication.

Brineura's list price is $27,000 per biweekly infusion, or $702,000 per year. The company said the drug's net price will be $486,000 after deducting mandatory government discounts and rebates, and factoring in patient compliance. Brineura will be available in June, BioMarin said...