BioMarin announced the submission of a Biologics License Application (BLA) to the FDA for Vimizim (BMN-110, elosulfase alfa), an enzyme replacement therapy under evaluation for the treatment of patients with rare lysosomal storage disorder Mucopolysaccaridosis Type IVA (MPS IVA), also called Morquio A Syndrome. Vimizim was formerly referred to as GALNS. The submission was based on positive results from a Phase 3 pivotal study in patients with MPS IVA.
MPS IVA is a disease characterized by deficient activity of N-acetylgalactosamine-6-sulfatase (GALNS) causing excessive lysosomal storage of glycosaminoglycans such as keratan sulfate and chondroitin sulfate. This excessive storage causes a systemic skeletal dysplasia, short stature, and joint abnormalities, which limit mobility and endurance.
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