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.

For more information call (877) 695-8826 or visit www.bmrn.com.