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alpha-glucosidase (CHO) (Genzyme).

Robin H Lachmann1

  • 1University of Cambridge, Department of Medicine, Box 157, Addenbrooke's Hospital, Hills Road, Cambridge CB2 2QQ, UK. rhl20@cam.ac.uk

Current Opinion in Investigational Drugs (London, England : 2000)
|November 13, 2004
PubMed
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Genzyme is developing recombinant human alpha-glucosidase for Pompe disease treatment. Clinical trials in adults were completed, with regulatory filings planned in Europe, the US, and Japan.

Area of Science:

  • Biotechnology
  • Genetics
  • Pharmacology

Background:

  • Pompe disease is a rare genetic disorder.
  • Enzyme replacement therapy is a potential treatment approach.

Purpose of the Study:

  • To evaluate recombinant human alpha-glucosidase as a treatment for Pompe disease.
  • To assess the safety and efficacy of the investigational therapy.

Main Methods:

  • Development of recombinant human alpha-glucosidase using mammalian cell culture.
  • Completion of enrollment in two clinical trials and an observational study in adult patients.
  • Monitoring of patient outcomes and adverse events.

Main Results:

  • Enrollment completed for adult Pompe disease clinical trials by July 2004.

Related Experiment Videos

  • Observational study in adults also completed enrollment.
  • Data collection and analysis ongoing.

    • Conclusions:

    • Recombinant human alpha-glucosidase shows promise as a Pompe disease therapy.
    • Regulatory submission planning is underway in key global markets.
    • Further data analysis will inform regulatory filings.