BioMarin Tests FDASIA’s Pediatric Voucher Waters With <em>Vimizim</em> Approval

BioMarin Tests FDASIA’s Pediatric Voucher Waters With Vimizim Approval

24 Feb 2014

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.

FDA’s decision to award BioMarin Pharmaceutical Inc. the first rare pediatric disease priority review voucher for Vimizim (elosulfase alfa) suggests the agency is taking a flexible approach to implementing the FDA Safety and Innovation Act incentive program.

Vimizim is broadly indicated for all patients with Morquio A syndrome – a rare, autosomal recessive lysosomal storage disease also...