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Cerdelga (ELIGLUSTAT)
Cerdelga works by inhibiting the enzyme ceramide glucosyltransferase, which is involved in the production of glucosylceramide.
Cerdelga (Eliglustat) is a small molecule glucosylceramide synthase inhibitor developed by Genzyme Corp. It targets ceramide glucosyltransferase to treat glucosylceramide beta-glucosidase deficiency. Cerdelga was FDA-approved in 2014 and is currently owned by Genzyme Corp. The drug is available as a generic product with multiple manufacturers. Key safety considerations include its half-life of 7.7 hours.
At a glance
| Generic name | ELIGLUSTAT |
|---|---|
| Sponsor | Sanofi |
| Drug class | Glucosylceramide Synthase Inhibitor [EPC] |
| Target | Ceramide glucosyltransferase |
| Modality | Small molecule |
| Therapeutic area | Metabolic |
| Phase | FDA-approved |
| First approval | 2014 |
| Annual revenue | 400 |
Mechanism of action
Gaucher disease is caused by deficiency of the lysosomal enzyme acid -glucosidase. Acid -glucosidase catalyzes the conversion of the sphingolipid glucocerebroside into glucose and ceramide. The enzymatic deficiency causes an accumulation of glucosylceramide (GL-1) primarily in the lysosomal compartment of macrophages, giving rise to foam cells or Gaucher cells.The clinical features of this lysosomal storage disorder (LSD) are reflective of the accumulation of Gaucher cells in the reticuloendothelial system (liver, spleen, bone marrow, and other organs). The accumulation of Gaucher cells in the liver, spleen, and bone marrow leads to organomegaly and skeletal disease. Presence of Gaucher cells in the bone marrow and spleen leads to clinically significant anemia and thrombocytopenia.CERDELGA is specific inhibitor of glucosylceramide synthase (IC50=10 ng/mL) and acts as substrate reduction therapy for GD1 by reducing the production of GL-1. By reducing GL-1 product
Approved indications
- Glucosylceramide beta-glucosidase deficiency
Common side effects
- Fatigue
- Headache
- Nausea
- Diarrhea
- Back pain
- Pain in extremity
- Upper abdominal pain
- Dizziness
- Asthenia
- Cough
- Dyspepsia
- Gastroesophageal reflux disease
Key clinical trials
- GSL Synthetase Inhibitor Plus Immune Checkpoint Inhibitor and/or Regorafenib in Previously Treated pMMR/MSS CRC. (PHASE2)
- Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3 (PHASE3)
- GSL Synthetase Inhibitor Eliglustat Combined With CD30 Target Immunotherapy for the Treatment of of CD30+ Lymphoma (PHASE1,PHASE2)
- GSL Synthetase Inhibitor Plus GM-CSF and/ or Immune Checkpoint Inhibitor in Previously Treated High-Risk Neuroblastoma. (EARLY_PHASE1)
- Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3 (PHASE2)
- 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)
- GSL Synthetase Inhibitor in Combination With Immune Checkpoint Inhibitor in Previously Treated Blood and Solid Tumor (PHASE1)
Patents
| Patent | Expiry | Type |
|---|---|---|
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 |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
| SEC EDGAR | Revenue + earnings |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Cerdelga CI brief — competitive landscape report
- Cerdelga updates RSS · CI watch RSS
- Sanofi portfolio CI