BioMarin's Brineura Approval Shows FDA's Open Door For Orphan Drugs
Filing supporting Brineura was supported by a tiny, single-arm study and had no advisory committee review, the latest sign of the US regulatory agency's willingness to speed orphan drugs for serious pediatric diseases.
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Priority Review Voucher Sales Prices Level Off In 2017
BioMarin's sale of voucher earned with approval of rare pediatric disease drug Brineura the fourth publicly disclosed voucher sale this year with a price tag between $125m and $150m.