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Drug

Eliglustat

Established medicine
ResearchOngoing clinical research

Also known as Cerdelga, Eliglustatum.

RxNorm1547220ChEMBLCHEMBL2110588ATCA16AX10UNIIDR40J4WA67
1
Active clinical trials
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Single-arm Exploratory Trial of GSL Synthetase Inhibitor Plus Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) and/ or Immune Checkpoint Inhibitor in Advanced/Metastatic Neuroblastoma.

Clinical trial2025-07-29Results expected Q3 2026 · ClinicalTrials.gov

Phase ⅠStudy of GSL Synthetase Inhibitor in Combination With Immune Checkpoint Inhibitor in Treating Patients With Advanced Relapsed or Refractory Hematological Malignancies and Previously Treated Solid Tumors

Clinical trial2023-11-30Completed · ClinicalTrials.gov

Evaluation of the Safety in the Combination Usage of Cerdelga and Cerezyme in Type III Gaucher Disease Patients and the Efficacy on Soft Tissue Diseases.

Clinical trial2020-09-11Completed · ClinicalTrials.gov

A Randomized, Three-Period Crossover Study of Single and Repeated Doses for Three Different Strengths of Eliglustat in Healthy Adult, CYP2D6 Extensive and Poor Metabolizers

Clinical trial2018-03-26Completed · ClinicalTrials.gov

An Open-label Two-stage Pharmacokinetic and Tolerability Study of Eliglustat Tartrate Given as a Single Dose in Subjects With Mild, Moderate and Severe Renal Impairment, and in Matched Subjects With Normal Renal Function

Clinical trial2017-01-01Completed · ClinicalTrials.gov

An Open-label Pharmacokinetic and Tolerability Study of Eliglustat Tartrate Given as a Single Dose in Subjects With Mild and Moderate Hepatic Impairment, and in Matched Subjects With Normal Hepatic Function

Clinical trial2016-12-01Completed · ClinicalTrials.gov

A Single-blind, Randomized, Unbalanced Crossover Design With 5 Vehicles, 5 Periods, and 5 Sequences, Repeated-doses (With no Ingestion) Study to Assess the Palatability of Eliglustat Prototype Liquid Formulations in Healthy Subjects

Clinical trial2015-05-01Completed · ClinicalTrials.gov

Approval: Cerdelga (EMA)

Regulatory2015-01-19EMA

An Open-label Study to Assess the Absorption Through the Mouth After Three Repeated 50 mg Doses of Eliglustat Solution, Separated by 2-hour Intervals, Held in the Mouth for 30 Seconds With Swishing But Without Ingestion, in Healthy Subjects

Clinical trial2014-09-21Completed · ClinicalTrials.gov

A Single-site, Open-label, Fixed-sequence Phase 1 Study Evaluating the Effect of Eliglustat (Genz-112638) on the Pharmacokinetics, Safety and Tolerability of Metoprolol in Healthy Adult Subjects.

Clinical trial2012-07-01Completed · ClinicalTrials.gov

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Eliglustat
Aliases & brands
CerdelgaEliglustatum
RxNorm CUI
1547220
ChEMBL ID
CHEMBL2110588
ATC codes
A16AX10
UNII
DR40J4WA67
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2015-01-19
Latest approval
2015-01-19
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2015-01-19Approval
Approval: Cerdelga (EMA)
Indication: AdultsCerdelga is indicated for the long-term treatment of adult patients with Gaucher disease type 1 (GD1), who are CYP2D6 poor metabolisers (PMs), intermediate metabolisers… Show full indication

AdultsCerdelga is indicated for the long-term treatment of adult patients with Gaucher disease type 1 (GD1), who are CYP2D6 poor metabolisers (PMs), intermediate metabolisers (IMs) or extensive metabolisers (EMs).Paediatric population (from 6 to < 18 years of age) weighing ≥ 15 kgCerdelga is indicated for paediatric patients with GD1 who are 6 years and older with a minimum body weight of 15 kg, who are stable on enzyme replacement therapy (ERT), and who are CYP2D6 PMs, IMs or EMs. 

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

9 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
9
registered trials across all phases
LATEST COMPLETION 2023
1
Active studies
1
Recruiting
1
Late-stage (III+)
8
Completed
PHASE DISTRIBUTIONn = 9
Early Phase 11Phase 17Phase N / A1

Recent completions

Trials that read out recently, adding to the completed evidence base.

Phase ⅠStudy of GSL Synthetase Inhibitor in Combination With Immune Checkpoint Inhibitor in Treating Patients With Advanced Relapsed or Refractory Hematological Malignancies and Previously Treated Solid Tumors

CompletedPH IChinese PLA General Hospitaln=312023

Evaluation of the Safety in the Combination Usage of Cerdelga and Cerezyme in Type III Gaucher Disease Patients and the Efficacy on Soft Tissue Diseases.

CompletedN/ANational Taiwan University Hospitaln=42020

A Randomized, Three-Period Crossover Study of Single and Repeated Doses for Three Different Strengths of Eliglustat in Healthy Adult, CYP2D6 Extensive and Poor Metabolizers

CompletedPH ISanofin=182018

An Open-label Two-stage Pharmacokinetic and Tolerability Study of Eliglustat Tartrate Given as a Single Dose in Subjects With Mild, Moderate and Severe Renal Impairment, and in Matched Subjects With Normal Renal Function

CompletedPH ISanofin=322017

An Open-label Pharmacokinetic and Tolerability Study of Eliglustat Tartrate Given as a Single Dose in Subjects With Mild and Moderate Hepatic Impairment, and in Matched Subjects With Normal Hepatic Function

CompletedPH IGenzyme, a Sanofi Companyn=242016

A Single-blind, Randomized, Unbalanced Crossover Design With 5 Vehicles, 5 Periods, and 5 Sequences, Repeated-doses (With no Ingestion) Study to Assess the Palatability of Eliglustat Prototype Liquid Formulations in Healthy Subjects

CompletedPH IGenzyme, a Sanofi Companyn=82015
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.