FDA Advisory Committee recommends Vimizim (BioMarin) for Morquio A syndrome

BioMarin Pharmaceutical Inc. announced that the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) of the FDA voted on 19 November 2013, in favour of approval of Vimizim (elosulfase alfa) for the treatment of Morquio A syndrome, also called Mucopolysaccharidosis Type IVA (MPS IVA). Of the 21 panel members, 19 voted in favour of approval of Vimizim for use in all MPS IVA patients, 1 voted in favour of approval for a subgroup of MPS IVA patients, and one panel member voted to not recommend approval.

Vimizim is an investigational enzyme replacement therapy for the treatment of patients with the lysosomal storage disorder Morquio A syndrome. Morquio A syndrome is an ultra-rare, severely debilitating disease that affects an estimated 3,000 patients in the developed world. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of February 28, 2014, for completion of its review of the Biologics License Application (BLA) for Vimizim.