Drug class

Glucosylceramide Synthase (GCS) Inhibitors

Glucosylceramide synthase (GCS) inhibitors are a therapeutic class of small-molecule agents that function via substrate reduction therapy. Read more about Glucosylceramide Synthase (GCS) Inhibitors

9 drugs in this class 8 manufacturers

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Drugs in Glucosylceramide Synthase (GCS) Inhibitors

Showing 1–9 of 9 drugs

ZAVESCA (miglustat)

GLUCOSYLCERAMIDE SYNTHASE (GCS) INHIBITORS · GAUCHER'S DISEASE
ACTELION PHARMA

MIGLUSTAT

GLUCOSYLCERAMIDE SYNTHASE (GCS) INHIBITORS · GAUCHER'S DISEASE
COTHERIX, INC.

OPFOLDA (miglustat)

GLUCOSYLCERAMIDE SYNTHASE (GCS) INHIBITORS · LYSOSOMAL ALPHA-1,4-GLUCOSIDASE DEFICIENCY
AMICUS THERAPEU

YARGESA (miglustat)

GLUCOSYLCERAMIDE SYNTHASE (GCS) INHIBITORS · GAUCHER'S DISEASE
EDENBRIDGE PHAR

MIGLUSTAT

GLUCOSYLCERAMIDE SYNTHASE (GCS) INHIBITORS · GAUCHER'S DISEASE
ANI PHARMACEUTI

MIGLUSTAT

GLUCOSYLCERAMIDE SYNTHASE (GCS) INHIBITORS · GAUCHER'S DISEASE
EDENBRIDGE/DEXC

YARGESA (miglustat)

GLUCOSYLCERAMIDE SYNTHASE (GCS) INHIBITORS · GAUCHER'S DISEASE
EDENBRIDGE/DEXC

MIGLUSTAT

GLUCOSYLCERAMIDE SYNTHASE (GCS) INHIBITORS · GAUCHER'S DISEASE
ZYDUS PHARMACEU
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About Glucosylceramide Synthase (GCS) Inhibitors.

Glucosylceramide synthase (GCS) inhibitors are a therapeutic class of small-molecule agents that function via substrate reduction therapy.

They act by selectively inhibiting UDP-glucose:ceramide glucosyltransferase, the rate-limiting enzyme responsible for catalyzing the initial step of glycosphingolipid biosynthesis.

By decreasing the production of glucosylceramide, these agents rebalance glycolipid metabolism and prevent pathological cellular accumulation.

Clinically, they are primarily approved and utilized for the management of type 1 Gaucher disease, serving as an effective oral alternative or adjunct for patients unsuitable for or unable to receive enzyme replacement therapy.