Drugs / Eliglustat
Eliglustat
also known as
Genz-112638 · Eliglustat tartrate · Cerdelga · Eliglustat (as tartrate) · Eliglustat hemitartrate · Eliglustat tartrate component of cerdelga · Eliglustat(Cerdelga) · GENZ 99067 · +1 more
Small molecule targets UGCG via inhibition
Regulatory milestones approvals, filings & regulatory actions · 3 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Label expansion | US (FDA) | CERDELGA | — | 2018-08-29 | fda.gov ↗ |
| Approved | EU (EMA) | Cerdelga | — | 2015-01-19 | europa.eu ↗ |
| Approved | US (FDA) | CERDELGA | — | 2014-08-19 | fda.gov ↗ |
Trials 20
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 01 trial | ||||||
| Phase 0 | NCT07090538 | Aug 2025 → Aug 2026 overdue | neuroblastoma | Chinese PLA General Hospital | Not yet recruiting | No outcome recorded |
| Phase 110 trials | ||||||
| Phase 1 | NCT04944888 | Jul 2021 → Nov 2023 | cancer, hematopoietic and lymphoid system neoplasm | Chinese PLA General Hospital | Completed | No outcome recorded |
| Phase 1 | NCT06188325 | Jan → Mar 2018 | Gaucher disease | Sanofi | Completed | No outcome recorded |
| Phase 1 | NCT02536937 | Sep 2015 → Jan 2017 | Gaucher disease | Sanofi | Completed | No outcome recorded |
| Phase 1 | NCT02536911 | Sep 2015 → Dec 2016 | Gaucher disease | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 1 | NCT02422654 | Apr → May 2015 | Gaucher disease | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 1 | NCT06193304 | Aug → Sep 2014 | Gaucher disease | Sanofi | Completed | No outcome recorded |
| Phase 1 | NCT01659944 | May → Jun 2012 | — | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 1 | NCT01452542 | Oct → Nov 2011 | — | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 1 | NCT01357811 | Aug → Nov 2011 | — | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 1 | NCT06143904 | Jun → Jul 2009 | Gaucher disease type I | Sanofi | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT07138547 | Dec 2025 → Aug 2028 expected | lymphoma | Chinese PLA General Hospital | Recruiting | No outcome recorded |
| Phase 23 trials · 1 met primary | ||||||
| Phase 2 | NCT06558773 | Mar 2026 → Mar 2027 expected | colorectal cancer | Chinese PLA General Hospital | Recruiting | No outcome recorded |
| Phase 2 | NCT06523517 | Aug 2024 → Jul 2025 overdue | Gaucher disease | Peking Union Medical College Hospital | Not yet recruiting | No outcome recorded |
| Phase 2 | NCT00358150 | Jun 2006 → Aug 2009 | Gaucher disease type I | Genzyme, a Sanofi Company | Completed | Met primary |
| Phase 34 trials · 2 met primary · 1 missed | ||||||
| Phase 3 | NCT02536755 | Oct 2015 → Jun 2021 | Gaucher disease | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 3 | NCT01074944 | Jun 2010 → Oct 2015 | Gaucher disease | Genzyme, a Sanofi Company | Completed | Missed primary |
| Phase 3 | NCT00891202 | Nov 2009 → Jul 2012 | Gaucher disease type I | Genzyme, a Sanofi Company | Completed | Met primary |
| Phase 3 | NCT00943111 | Sep 2009 → Nov 2012 | Gaucher disease type I | Genzyme, a Sanofi Company | Completed | Met primary |
| Phase not applicable1 trial | ||||||
| — | NCT03519646 | Apr 2018 → Sep 2020 | Gaucher disease type III | National Taiwan University Hospital | Completed | No outcome recorded |
Evidence & citations 14 cited values
Every value below carries the sentence it was read from. 22 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Eliglustat | ClinicalTrials.gov intervention name — accepted as the source's own label NCT01357811 ↗ |
| Known as | Cerdelga | ChEMBL registry synonym — accepted as the source's own label CHEMBL2110588 ↗ |
| Known as | Eliglustat (as tartrate) | ChEMBL registry synonym — accepted as the source's own label CHEMBL2110588 ↗ |
| Known as | Eliglustat hemitartrate | ChEMBL registry synonym — accepted as the source's own label CHEMBL2110588 ↗ |
| Known as | Eliglustat tartrate | “The primary objective of this study was to evaluate the efficacy and safety of once daily (QD) versus twice daily (BID) dosing of eliglustat tartrate (Genz-112638) in...” NCT01074944 ↗2“Eliglustat tartrate (Genz-112638), a specific inhibitor of glucosylceramide synthase, is under development as an oral substrate reduction therapy for Gaucher disease type 1 (GD1).” PMID 20439622 ↗ May 2010 |
| Known as | Eliglustat tartrate component of cerdelga | ChEMBL registry synonym — accepted as the source's own label CHEMBL2110588 ↗ |
| Known as | Eliglustat(Cerdelga) | “Eliglustat(Cerdelga)” NCT06558773 ↗ |
| Known as | GENZ 99067 | ChEMBL registry synonym — accepted as the source's own label CHEMBL2110588 ↗ |
| Known as | Genz-112638 | “A Single-site, Open-label, Fixed-sequence Phase 1 Study Evaluating the Effect of Eliglustat (Genz-112638) on the Pharmacokinetics and Safety and Tolerability of Digoxin in...” NCT01357811 ↗3“Eliglustat tartrate (Genz-112638), a specific inhibitor of glucosylceramide synthase, is under development as an oral substrate reduction therapy for Gaucher disease type 1 (GD1).” PMID 20439622 ↗ May 2010 “single dose GenZ-112638 oral capsules Day 8” NCT06143904 ↗ |
| Known as | GZ385660 | “Eliglustat, GZ385660” NCT02536755 ↗ |
| Action | Inhibit | “a specific inhibitor of glucosylceramide synthase” PMID 20439622 ↗ May 20101“Ceramide glucosyltransferase inhibitor” CHEMBL2110588 ↗ |
| Modality | Small molecule | “Eliglustat tartrate is a small molecule developed as an oral therapy” NCT00891202 ↗ |
| Route | Oral | “administered twice daily by mouth at 50- or 100-mg doses” PMID 20439622 ↗ May 2010 |
| Target | UGCG | “Ceramide glucosyltransferase inhibitor” CHEMBL2110588 ↗ |