FDA committee recommends approval for BioMarin's Vimizim

20 November 2013

A US Food and Drug Administration panel has voted in favor of approval of Vimizim (BMN-110, elosulfase alfa) for the treatment of Morquio A syndrome from USA-based rare disease drug developer BioMarin Pharmaceuticals (Nasdaq: BMRN).

The Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) voted 20 to one in favor for use in all Morquio A syndrome patients, also called Mucopolysaccharidosis Type IVA (MPS IVA). One voted in favor of approval for a subgroup of MPS IVA patients, and one panel member voted to not recommend approval. The committee additionally voted 16-five that no safety concerns were associated with the enzyme replacement therapy.

Could achieve sales of over $500 million in 2018

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