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Drugs in R & D

, Volume 6, Issue 5, pp 312–315 | Cite as

Galsulfase

Arylsulfatase B, BM 102, Recombinant Human Arylsulfatase B, Recombinant Human N-Acetylgalactosamine-4-Sulfatase, rhASB
Adis R&D Profile

Abstract

Galsulfase [Aryplase™, arylsulfatase B, BM 102, Naglazyme™, rhASB, recombinant human N-acetylgalactosamine-4-sulfatase, recombinant human arylsulfatase B] is under development with BioMarin Pharmaceutical as an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) VI.

MPS VI (also known as Maroteaux-Lamy syndrome) is a progressive, debilitating genetic disease resulting in early death. Patients with MPS VI have a deficiency in the arylsulfatase B (ASB) enzyme that is essential for the progressive breakdown of certain complex carbohydrates. The deficiency in ASB results in the build-up of carbohydrate residues in the lysosomes in all cells of the body. Patients are usually diagnosed at 6–24 months of age, and the symptoms include deceleration of growth, enlarged liver and spleen, skeletal and joint deformities, and upper airway obstruction. Patients do not survive past 20–30 years of age in the more severe cases, but may live longer with the milder cases,...

Keywords

Enzyme Replacement Therapy Arylsulfatase Orphan Drug Status Biologics License Application Galsulfase 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.

References

  1. 1.
    BioMarin Pharmaceutical Inc. BioMarin Submits U.S. Biologics License Application for Marketing Authorization of Aryplase for MPS VI. Media Release: 29 Nov 2004. Available from URL: http://www.bmrn.com
  2. 2.
    BioMarin Pharmaceutical Inc. FDA Accepts and Grants Six- Month Review for BioMarin’s Marketing Application for rhASB for MPS VI. Media Release: 1 Feb 2005. Available from URL: http://www.bmrn.com Google Scholar
  3. 3.
    BioMarin Pharmaceutical Inc. BioMarin Presents Positive Phase II Data on Aryplase for MPS VI. Media Release: 14 Mar 2003. Available from: URL: http://www.biomarinpharm.com Google Scholar
  4. 4.
    BioMarin Pharmaceutical Inc. BioMarin Announces Positive Phase 3 Data on Aryplase(TM) for MPS VI. Media Release: 3 Jun 2004. Available from URL: http://www.bmrn.com Google Scholar
  5. 5.
    BioMarin Pharmaceutical Inc. BioMarin Announces FDA Approval for Naglazyme. Media Release 1 Jun 2005. Available from URL: http://www.bmrn.com Google Scholar
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    BioMarin Pharmaceutical Inc. BioMarin Announces U.S. Launch of Naglazyme for MPS VI. Media Release: 21 Jun 2005. Available from URL: http://www.bmrn.com Google Scholar
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    BioMarin Pharmaceutical Inc. BioMarin Presents Positive Long-Term Results from Ongoing Phase 1 and Phase 2 Clinical Studies of Aryplase for MPS VI. Media Release: 5 Nov 2003. Available from URL: http://www.biomarinpharm.com Google Scholar
  8. 8.
    BioMarin Pharmaceutical Inc. BioMarin’s Phase 3 Extension Study of rhASB in MPS VI Demonstrates Additional Improvements in Endurance. Media Release: 9 Mar 2005. Available from URL: http://www.bmrn.com Google Scholar

Copyright information

© Adis Data Information BV 2005

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