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Eliglustat GZ385660
Eliglustat is a glucosylceramide synthase (GCS) inhibitor that reduces the production of glucosylceramide, a lipid that accumulates in Gaucher disease.
Eliglustat is a glucosylceramide synthase (GCS) inhibitor that reduces the production of glucosylceramide, a lipid that accumulates in Gaucher disease. Used for Gaucher disease type 1.
At a glance
| Generic name | Eliglustat GZ385660 |
|---|---|
| Also known as | Cerdelga |
| Sponsor | Sanofi |
| Drug class | Glucosylceramide synthase inhibitor |
| Target | Glucosylceramide synthase (GCS) |
| Modality | Small molecule |
| Therapeutic area | Rare/Genetic Disorders |
| Phase | Phase 3 |
Mechanism of action
Eliglustat works by inhibiting glucosylceramide synthase, the enzyme responsible for synthesizing glucosylceramide from glucose and ceramide. By blocking this enzyme, the drug reduces the accumulation of glucosylceramide in macrophages and other cells affected in Gaucher disease, thereby reducing organomegaly and other disease manifestations. This substrate reduction therapy approach addresses the underlying pathophysiology of lysosomal storage disorders.
Approved indications
- Gaucher disease type 1
Common side effects
- Diarrhea
- Tremor
- Headache
- Fatigue
Key clinical trials
- Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3 (PHASE3)
- A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor Metabolizers (PHASE1)
- A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth (PHASE1)
- A Study to Evaluate Absolute Bioavailability, Absorption, Metabolism, and Excretion of Genz-112638 in Healthy Male Participants (PHASE1)
- Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies (PHASE3)
- A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate (PHASE1)
- A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate (PHASE1)
- Taste Evaluation of Different Liquid Formulations With Eliglustat (PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Eliglustat GZ385660 CI brief — competitive landscape report
- Eliglustat GZ385660 updates RSS · CI watch RSS
- Sanofi portfolio CI