eliglustat

Trade name: cerdelga

Small moleculeapprovedOrphan Drug ^FDA

Approved

Aug 19, 2014

Eliglustat, marketed by Genzyme as CERDELGA, is a glucosylceramide synthase inhibitor indicated for the long-term treatment of type 1 Gaucher disease who are CYP2D6 extensive metabolizers, intermediate metabolizers, or poor metabolizers (PMs) as detected by an FDA-cleared test. — NCATS

Clinical trial activity

21 trials · 5 clinical orgs · 10 marketing orgs

Phase 1

Phase 2

Phase 3

Phase 4

Earliest trial started Jun 1, 2006 (NCT00358150)

Timeline

2000s

Jan 1, 2006
Genzyme — Earliest Phase 2 Sponsor(trial)
Sep 17, 2008
Orphan Drug Designation
Jan 1, 2009
Earliest Phase 1 Sponsor(trial)
Jan 1, 2009
Genzyme — Earliest Phase 3 Sponsor(trial)

2010s

Aug 19, 2014
Genzyme — Earliest FDA Approval
Aug 19, 2014
Genzyme — NDA Organization
Aug 19, 2014
Sanofi-Aventis — NDA Secondary Org

2020s

Sep 8, 2021
Appco Pharma — Marketing Organization
Sep 8, 2021
Aizant — Marketing Organization
Dec 27, 2021
Teva — Marketing Organization
Aug 17, 2022
Dr. Reddy's Laboratories — Marketing Organization
May 2, 2023
ZENARA — Marketing Organization
May 2, 2023
Upsher-Smith Laboratories — Marketing Organization
Jul 10, 2024
Apotex — Marketing Organization
Dec 10, 2024
Cipla — Marketing Organization

Indications

Approved for

Gaucher Disease

Studied for

Mechanism of action

Ceramide glucosyltransferaseINHIBITOR
UGCG inhibitor
- Ceramide glucosyltransferase (UGCG) ↗

Approval history

approvedPriority reviewAug 19, 2014

Chemistry & pharmacology

Loading structure…

SMILES

CCCCCCCC(=O)N[C@H](CN1CCCC1)[C@H](O)c1ccc2c(c1)OCCO2

Mol. weight: 404.55 g/mol
Lipinski Ro5: Pass
Rule of 3: No
Chirality: Single Stereoisomer
Inorganic: No
Polymer: No
Delivery: Oral
Availability: Prescription Only

Oral

Yes

Parenteral

Topical

Sources

Also known as

cerdelga
cerdelga
cerdelga
cerdelga
cerdelga
cerdelga
cerdelga
eliglustat
eliglustat
eliglustat
eliglustat
eliglustat
eliglustat
eliglustat
eliglustat
eliglustat
eliglustat
eliglustat tartrate
eliglustat tartrate
eliglustat tartrate
eliglustat tartrate
eliglustat tartrate
eliglustat tartrate
eligustat
genz-112638
genz-112638
genz-112638
genz-112638
genz-99067
n-[(1r,2r)-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-(1-pyrrolidinyl)-2-propanyl]octanamide
n-[(1r,2r)-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-(1-pyrrolidinyl)-2-propanyl]octanamide

Clinical trials

NCT ID	Phase	Start date	Sponsor(s)
NCT07138547	Phase 1/Phase 2 (Phase 2)	Sep 1, 2025	People's Liberation Army of China
NCT07090538	Early Phase 1 (Phase 1)	Aug 1, 2025	People's Liberation Army of China
NCT06558773	Phase 2	Sep 1, 2024	People's Liberation Army of China
NCT06523517	Phase 2	Aug 1, 2024	Chinese Academy of Medical Sciences
NCT04944888	Phase 1	Jul 1, 2021	People's Liberation Army of China
NCT03519646	N/A	May 31, 2018	National Taiwan University, Sanofi
NCT03485677	Phase 3	Apr 11, 2018	Sanofi
NCT06188325	Phase 1	Jan 1, 2018	Sanofi
NCT02536755	Phase 3	Oct 28, 2015	Genzyme, Sanofi
NCT02536937	Phase 1	Sep 1, 2015	Sanofi
NCT02536911	Phase 1	Sep 1, 2015	Genzyme, Sanofi
NCT02422654	Phase 1	Apr 1, 2015	Genzyme, Sanofi
NCT06193304	Phase 1	Aug 25, 2014	Sanofi
NCT01659944	Phase 1	May 1, 2012	Genzyme
NCT01452542	Phase 1	Oct 1, 2011	Genzyme
NCT01357811	Phase 1	Aug 1, 2011	Genzyme
NCT01074944	Phase 3	Jun 1, 2010	Genzyme
NCT00891202	Phase 3	Nov 1, 2009	Genzyme
NCT00943111	Phase 3	Sep 1, 2009	Genzyme
NCT06143904	Phase 1	Jun 3, 2009	Sanofi

Showing 20 of 21 trials

Page 1 / 2

Organizations

Research & Development (5)

Organization	Org type	Trials	As lead sponsor	Phases	Earliest year
Genzyme	For profit	10	10	3	2006
Sanofi	For profit	9	5	3	2009
People's Liberation Army of China	Government	4	4	2	2021
Chinese Academy of Medical Sciences	Academic/Hospital	1	1	1	2024
National Taiwan University	Academic/Hospital	1	1	1	2018

5 organizations

Page 1 / 1

Marketing (10)

Organization	Org type	Relationship	Date
Aizant	For profit	MKTG	Sep 8, 2021
Apotex	For profit	MKTG	Jul 10, 2024
Appco Pharma	For profit	MKTG	Sep 8, 2021
Cipla	For profit	MKTG	Dec 10, 2024
Dr. Reddy's Laboratories	For profit	MKTG	Aug 17, 2022
Genzyme	For profit	NDA	Aug 19, 2014
Sanofi-Aventis	For profit	NDA2	Aug 19, 2014
Teva	For profit	MKTG	Dec 27, 2021
Upsher-Smith Laboratories	For profit	MKTG	May 2, 2023
ZENARA	For profit	MKTG	May 2, 2023