FDA Grants Priority Review, Biologics License Application to Metabolic Disease

Spinal Tech

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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