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Eliglustat: first global approval.

Raewyn M Poole1

  • 1Springer, Private Bag 65901, Mairangi Bay 0754, Auckland, New Zealand, dru@adis.com.

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|September 21, 2014
PubMed
Summary
This summary is machine-generated.

Eliglustat, an oral glucosylceramide synthase inhibitor, effectively treats Gaucher disease type 1 by reducing lipid accumulation. This marks a significant milestone in adult therapeutic options for this rare genetic disorder.

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Area of Science:

  • Biochemistry
  • Pharmacology
  • Genetics

Background:

  • Gaucher disease type 1 is a genetic disorder characterized by glucosylceramide accumulation.
  • Current treatments may have limitations, necessitating novel therapeutic approaches.

Purpose of the Study:

  • To summarize the development of eliglustat, a glucosylceramide synthase inhibitor.
  • To highlight the milestones leading to eliglustat's approval for Gaucher disease type 1.

Main Methods:

  • Development of eliglustat as a small-molecule oral inhibitor.
  • Inhibition of glucosylceramide synthase to reduce lipid deposition.

Main Results:

  • Eliglustat reduces glucosylceramide accumulation in organs like the liver, spleen, and bone marrow.
  • Eliglustat received its first global approval in the US for adult patients.

Conclusions:

  • Eliglustat represents a new oral treatment option for Gaucher disease type 1.
  • The drug's development signifies progress in managing this lysosomal storage disorder.