Phase 3 study of GALNS for the treatment of Morquio A Syndrome

BioMarin Pharmaceuticals announced that it has initiated a Phase 3 trial for N-acetylgalactosamine 6-sulfatase (GALNS or BMN 110), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome. The trial is a randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of GALNS in about 160 patients with MPS IVA. The study will explore doses of 2mg/kg/week and 2mg/kg/every other week for a treatment period of 24 weeks. The primary endpoint is the 6-minute walk test, and the secondary endpoints are the 3-minute stair climb test and urine keratan sulfate concentration.  

MPS IVA or Morquio A Syndrome, is a disease characterized by deficient activity of N-acetylgalactosamine 6-sulfatase (GALNS) causing excessive lysosomal storage of keratan sulfate (KS). This excessive storage causes a systemic skeletal dysplasia, short stature, and joint abnormalities, which limit mobility and endurance.  BioMarin’s GALNS therapy is intended as an enzyme replacement to treat MPS IVA.

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