BioMarin's Vimizim BLA accepted by FDA and MAA validated by EMA

US drugmaker BioMarin Pharmaceutical (Nasdaq: BMRN) says that the Food and Drug Administration  has accepted for review the Biologics License Application 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 (MPS IVA), also called Morquio A Syndrome.

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 (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.