FDA cleared BioMarin Pharmaceutical Inc.’s Morquio A syndrome treatment Vimizim (elosulfase alfa) Feb. 14 with a broad label that places no limits on the enzyme replacement therapy’s use either by baseline disease severity or age – despite agency questions about efficacy in patients with better mobility and a lack of efficacy and safety data in very young children.
Two weeks ahead of its Feb. 28 PDUFA deadline, FDA approved Vimizim, a hydrolytic lysosomal glycosaminoglycan-specific enzyme, as for the...
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