FDA Grants Priority Review, Biologics License Application to Metabolic Disease

Written by Heather Linder | May 31, 2013 | Print  |
The FDA has accepted BioMarin Pharmaceutical's biologics license application for Vimizim, an enzyme therapy that could be used to treat Mucopolysaccharidosis Type IVA or Morquio A Syndrome, according to Pipeline Review.Mucopolysaccharidosis is a metabolic disorder caused by missing or deficient enzymes needed to break down the keratan sulfate sugar chain. It is physically manifested through skeletal abnormalities, such as a flattening or curvature of the spine and dysplasia of the hips and knees.

Vimizim was also granted a priority review designation, a designation given to drugs that offer major treatment advances, according to the report.

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