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NAGLAZYME® (galsulfase) reduced urinary GAG levels

NAGLAZYME reduced urinary glycosaminoglycans

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  • Week 24: In patients treated with NAGLAZYME, mean urinary GAG levels were reduced by 75% at week 24.7

- Urinary GAG excretion was selected as a secondary efficacy variable based on the association of reduction in lysosomal storage with the reduction in urinary GAGs observed during recombinant arylsulfatase B (rhASB) treatment of feline MPS VI.7,21,22

  • Week 96: In the group originally treated with NAGLAZYME, the reduction in GAG levels was sustained through week 96.16 From week 48 to week 96, no change in urinary GAG levels was observed in the NAGLAZYME group.20
  • Crossover: The placebo group began taking NAGLAZYME at week 24. At week 36, mean urinary GAG levels in this crossover group decreased to a level similar to patients originally in the NAGLAZYME treatment group.
  • Overall, 95% of patients showed at least a 50% reduction in urinary GAG levels after 72 weeks of treatment with NAGLAZYME.1

* One patient in the placebo group left the study for reasons unrelated to treatment. Patients receiving placebo were switched to NAGLAZYME in the trial extension period.7
†One patient failed to complete the assessment.20
‡Two patients failed to complete the assessment.20

 Next: Dosing and administration

References

  1. Wilcox WR. Lysosomal storage disorders: the need for better pediatric recognition and comprehensive care. J Pediatr. 2004;144(5 Suppl):S3–S14.
  1. Harmatz P, Giugliani R, Schwartz I, et al. Enzyme replacement therapy for mucopolysaccharidosis VI: a Phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study. J Pediatr. 2006;148:533-539.
  1. Wraith JE. The mucopolysaccharidoses: a clinical review and guide to management. Arch Dis Childhood. 1995;72:263–267.
  1. Harmatz RP. Mucopolysaccharidosis type VI. Emedicine. December 18, 2006. Available at: http://www.Emedicine.com/PED/topic1373.htm Accessed: November 4, 2008.
  1. Harmatz P, Ketteridge D, Giugliani R, et al. Direct comparison of measures of endurance, mobility, and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): results after 48 weeks in a phase 2 open-label clinical study of recombinant human N-acetylgalactosamine 4-sulfatase. Pediatrics. 2005;115:e681–e689.
  1. Harmatz P, Giugliani R, Schwartz IV, Guffon N, Teles EL, Miranda MC, et al. Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfastase. Mol Genet Metab. 2008. doi:10.1016/j.ymgme.2008.04.001.