BioMarin Tests FDASIA’s Pediatric Voucher Waters With Vimizim Approval
FDA clearance of the Morquio A syndrome treatment provides some insight into how the agency is implementing the 2012 priority review voucher program aimed at incentivizing development of treatments for rare pediatric diseases.
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Priority Review Voucher Not For Sale, Alexion Says After Strensiq Approval
Alexion plans to use voucher for one of its own products and might be targeting its next-generation version of blockbuster Soliris.
Low Survival To Adulthood Key To Vimizim Pediatric Priority Voucher
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.
Review Cost For Sanofi/Regeneron’s PCSK9 Inhibitor Remains Uncertain
Even after paying BioMarin $67.5 million for the voucher itself, the firms still don’t know how much FDA will charge for a chance to get Praluent to market faster.