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Few Clouds On High-Priced, Ultra-Orphan Drug Horizon

 
• By Mary Jo Laffler and Emily Hayes

With BioMarin's Brineura hitting the market with a $702,000 annual price tag, recent experience for other high-priced rare disease drugs supports the theory that payers will accept high costs for ultra-rare pediatric disease therapies.

Money Wave

Pricing has never really been an issue for rare disease drugs, given the low exposure payers have to the therapies and the significant benefits usually offered. But with ultra-orphan price tags ticking higher and higher and drug pricing a simmering political controversy in the US, it is worth checking on the experience of recent launches as BioMarin Pharmaceutical Inc. introduces its Batten disease therapy Brineura (cerliponase alfa) at just over $700,000.

See Related Story

For details of the Brineura approval, see BioMarin Expects 30% Markdown For Orphan Pediatric Drug Brineura.

Two antisense oligonucleotide therapies approved last year for different neurodegenerative conditions, Biogen/Ionis Pharmaceuticals Inc.'s Spinraza (nusinersen) for spinal...

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