BioCentury
ARTICLE | Clinical News

Vimizim elosulfase alfa regulatory update

May 5, 2014 7:00 AM UTC

BioMarin said the European Commission approved Vimizim elosulfase alfa to treat mucopolysaccharidosis IVA (MPS-IVA, Morquio's syndrome), a rare inherited lysosomal storage disease. The company has begun supplying the drug in the EU and said it is working with individual countries to establish Vimizim's price. In February, BioMarin launched the recombinant human N-acetylgalactosamine-6-sulfatase in the U.S. at a wholesale acquisition cost of $1,068 for a 5 mg vial. The recommended dose is 2 mg/kg once weekly. Including "discounts and expected compliance," the company said this translates to an average annual cost of about $380,000 for a typical 22.5 kg patient. ...