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Vimizim, elosulfase alfa (BMN-110) (formerly GALNS)

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Collapse Summary General Information
Company BioMarin Pharmaceutical Inc.
DescriptionRecombinant human N-acetylgalactosamine-6-sulfatase
Molecular Target
Mechanism of ActionEnzyme replacement therapy
Therapeutic ModalityBiologic: Enzyme
Latest Stage of DevelopmentMarketed
Standard IndicationMucopolysaccharidosis
Indication DetailsTreat mucopolysaccharidosis IVA (MPS IVA, Morquio's syndrome)
Regulatory Designation

U.S. - Orphan Drug (Treat mucopolysaccharidosis IVA (MPS IVA, Morquio's syndrome));
U.S. - Priority Review (Treat mucopolysaccharidosis IVA (MPS IVA, Morquio's syndrome));
EU - Orphan Drug (Treat mucopolysaccharidosis IVA (MPS IVA, Morquio's syndrome));
EU - Standard Review (Treat mucopolysaccharidosis IVA (MPS IVA, Morquio's syndrome));
Japan - Orphan Drug (Treat mucopolysaccharidosis IVA (MPS IVA, Morquio's syndrome));
Canada - Standard Review (Treat mucopolysaccharidosis IVA (MPS IVA, Morquio's syndrome))

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