BioMarin Pharmaceutical Inc. (BMRN) announced that the U.S. Food and Drug Administration or FDA has accepted for review the Biologics License Application or BLA for Vimizim (BMN-110, elosulfase alfa), an enzyme replacement therapy under evaluation for the treatment of patients with the rare lysosomal storage disorder Mucopolysaccharidosis Type IVA or MPS IVA, also called Morquio A Syndrome.
The company said that the FDA has granted priority review designation to Vimizim, which is granted to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists. During the initial review of the application, the FDA requested additional Chemistry, Manufacturing and Controls or CMC information.
The company provided the information as requested, and the FDA designated it as a major amendment to the application thus extending the PDUFA action date by three months. The extended PDUFA action date is February 28, 2014.
In the FDA's filing communication, the Agency informed the company that it is currently planning to hold an advisory committee meeting to discuss the application. No date has been set for this meeting.
by RTT Staff Writer
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