FDA approves Vimizim to treat rare congenital enzyme disorder

The US Food and Drug Administration on Friday (February 14) approved BioMarin Pharmaceutical’s (Nasdaq: BMRN) Vimizim (elosulfase alfa), the first FDA-approved treatment for Mucopolysaccharidosis Type IVA (Morquio A syndrome).

The approval comes as no surprise because, last fall, the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) voted 20 to one in favor for use if Vimizim in all Morquio A syndrome patients, also called Mucopolysaccharidosis Type IVA (MPS IVA), a rare, autosomal recessive lysosomal storage disease caused by a deficiency in N-acetylgalactosamine-6-sulfate sulfatase (GALNS; The Pharma Letter November 20, 2013).

Shipments of Vimizim to the distribution channels will commence immediately, and BioMarin will begin promotion of the drug in the USA immediately, the company said. BioMarin also noted that it has also submitted marketing applications for Vimizim in the European Union, Brazil, Australia, Canada, and Mexico.

Sales could exceed $500 million a year by 2018, say analysts