The FDA announced the approval of Vimizim (elosulfase alfa; BioMarin) injection for the treatment of Mucopolysaccharidosis Type IVA (Morquio A syndrome).

Vimizim is intended to provide the exogenous enzyme N-acetylgalactosamine-6-sulfatase (GALNS) that will be taken up into the lysosomes and increase the catabolism of the GAGs KS and C6S. Absence of this enzyme leads to problems with bone development, growth, and mobility.

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The safety and efficacy of Vimizim were established in a clinical study involving 176 patients with Morquio A syndrome, ranging from 5–57 years of age. Patients treated with Vimizim showed greater improvement in a 6-minute walk test than patients treated with placebo. In addition, patients treated with Vimizim walked 22.5m farther in 6 minutes compared to the patients who received placebo.

Vimizim will be available as a 1mg/mL concentrated solution for infusion in 5mL vials.

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