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Cerdelga, eliglustat tartrate (formerly Genz-112638)

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Company Sanofi
DescriptionCeramide analog that inhibits glucosylceramide synthase (GCS)
Molecular Target Glucosylceramide synthase (GCS)
Mechanism of ActionGlucosylceramide synthase (GCS) inhibitor
Therapeutic ModalitySmall molecule
Latest Stage of DevelopmentMarketed
Standard IndicationGaucher's disease
Indication DetailsTreat Gaucher's disease; Treat Type I Gaucher's disease
Regulatory Designation U.S. - Orphan Drug (Treat Type I Gaucher's disease);
U.S. - Priority Review (Treat Type I Gaucher's disease);
EU - Standard Review (Treat Type I Gaucher's disease);
Japan - Orphan Drug (Treat Type I Gaucher's disease)

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