Cerdelga

(Eliglustat)

Phase 3Small molecule

Cerdelga is a small molecule that works as a Glucosylceramide Synthase Inhibitor.[2] It is being investigated across 3 indications, with its most advanced program in Phase 3.[1]

Cerdelga is one of 71 small molecule programs targeting Neuroblastoma. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

NeuroblastomaActive, not recruiting

Gaucher Disease Type 1Active, not recruiting

Gaucher DiseaseActive, not recruiting

NeuroblastomaPhase 1

Active, not recruiting

Gaucher Disease Type 1Phase 2

Active, not recruiting

Gaucher DiseasePhase 3

Active, not recruiting

Competition

Competitive Landscape

237 competitors in Neuroblastoma

#DrugCompanyPhaseTrials

Leukine

Sanofi

Phase 3

18 2

Paraplatin

AAvyxa

Phase 3

38 3

Vepesid

AAvyxa

Phase 3

42 4

Cytoxan

BBaxter Healthcare Corporation

Phase 3

54 5

Camptosar

Pfizer

Phase 3

13 6

Tepadina

Alnylam Pharmaceuticals

Phase 3

12 7

Accutane

CConrado Roberto Hoffmann Filho

Phase 3

28 8

Alkeran

AApotex Inc.

Phase 3

28 9

Oncovin

EEastern Cooperative Oncology Group

Oncovin

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q3 2027

Phase 1Not yet recruiting

Neuroblastoma

NCT07090538

Clinical

Trials by Indication[1]

Gaucher Disease4 trials

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)

Completed· Sanofi logo

Sanofi·Phase 3·NCT00891202·40 enrolled

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)

Completed· Sanofi logo

Sanofi·Phase 3·NCT00943111·160 enrolled

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)

Completed· Sanofi logo

Sanofi·Phase 3·NCT01074944·170 enrolled

Show 1 more trial

Eliglustat on Gaucher Disease Type IIIB

Completed·NNational Taiwan University Hospital·N/A·NCT03519646·4 enrolled

Efficacy and Safety1 trial

A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients

Completed· Sanofi logo

Sanofi·Phase 2·NCT00358150·26 enrolled

Gaucher disease type 11 trial

Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3

Not yet recruiting·PPeking Union Medical College Hospital·Phase 2·NCT06523517·5 enrolled

Renal Impairment1 trial

A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate

Completed· Sanofi logo

Sanofi·Phase 1·NCT02536937·32 enrolled

Immune Checkpoint Inhibitor1 trial

GSL Synthetase Inhibitor in Combination With Immune Checkpoint Inhibitor in Previously Treated Blood and Solid Tumor

Completed·—·Phase 1·NCT04944888·31 enrolled

5 more indications

Neuroblastoma1 trial

GSL Synthetase Inhibitor Plus GM-CSF and/ or Immune Checkpoint Inhibitor in Previously Treated High-Risk Neuroblastoma.

Not yet recruiting·—·Phase 1·NCT07090538·10 enrolled

Absorption1 trial

A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth

Completed· Sanofi logo

Sanofi·Phase 1·NCT06193304·6 enrolled

Adult1 trial

Study to Evaluate the Effect of Eliglustat on the Pharmacokinetics, Safety and Tolerability of Metoprolol in Healthy Adults

Completed· Sanofi logo

Sanofi·Phase 1·NCT01659944·14 enrolled

Hepatic Impairment1 trial

A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate

Completed· Sanofi logo

Sanofi·Phase 1·NCT02536911·24 enrolled

Unspecified2 trials

Taste Evaluation of Different Liquid Formulations With Eliglustat

Completed· Sanofi logo

Sanofi·Phase 1·NCT02422654·8 enrolled

A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor Metabolizers

Completed· Sanofi logo

Sanofi·Phase 1·NCT06188325·18 enrolled

Regulatory

FDA Approval Timeline[4]

Aug 19, 2014

CERDELGA

NDA NDA205494

Sources

References

[1]
ClinicalTrials.gov — 14 trial records for Eliglustat. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Eliglustat compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Eliglustat. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA205494). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Cerdelga. U.S. Food & Drug Administration.
[6]
SEC EDGAR — Genzyme, a Sanofi Company company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Glucosylceramide Synthase Inhibitor[2]
Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: GGenzyme, a Sanofi Company

Patent Landscape[5]

Patent Cliff

Nov 24, 2030

Earliest patent expiration — generic entry possible after this date

Patent #Expires

7196205Jun 26, 2026

11458119Nov 24, 2030

10888547Jan 31, 2031

12465586Jun 5, 2032

10888544Dec 13, 2038

Cerdelga

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q3 2027

Trials by Indication[1]

Gaucher Disease4 trials

Efficacy and Safety1 trial

Gaucher disease type 11 trial

Renal Impairment1 trial

Immune Checkpoint Inhibitor1 trial

Neuroblastoma1 trial

Absorption1 trial

Adult1 trial

Hepatic Impairment1 trial

Unspecified2 trials

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

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