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BioMarin Expects 30% Markdown For Orphan Pediatric Drug Brineura

 
• By Emily Hayes

Fast US approval of ultra-rare pediatric disease drug Brineura (cerliponase alfa), with an annual list price of $702,000, is a victory for the company, but approval for restricted pediatric population compared to Europe raised questions.

BioMarin Pharmaceutical Inc. gave its Brineura (cerliponase alfa) a high sticker price of $702,000 per year for treatment of a life-threatening, ultra-rare pediatric brain condition, but the company expects for most patients it will cost $486,000 – almost 31% less – upon launch in June.

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