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Low Survival To Adulthood Key To Vimizim Pediatric Priority Voucher

 
• By Derrick Gingery

FDA says that Morquio A syndrome qualifies as a rare pediatric disease because most patients with a severe form of the disease will not survive into adulthood, paving the way for BioMarin to receive the first rare pediatric disease priority review voucher.

Morquio A syndrome may be seen in adults, but FDA classified it as a rare pediatric disease because it begins in childhood and many patients with a severe form of the syndrome do not survive to adulthood.

The decision meant that BioMarin Pharmaceutical Inc. was eligible for (and ultimately received) the first rare pediatric disease priority...

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