1. Pink Sheet

BioMarin's Brineura Approval Shows FDA's Open Door For Orphan Drugs

 
• By Emily Hayes and Sue Sutter

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.

Rapid development and FDA approval of BioMarin Pharmaceutical Inc.'s Brineura (cerliponase alfa) for a rare, life-threatening form of Batten disease afflicting children shows that despite some past controversy, the regulatory doors are still wide open for orphan drugs.

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