obeticholic acid

Trade name: ocaliva

Small moleculeapprovedOrphan Drug ^FDAFast Track ^FDA

Approved

May 27, 2016

Obeticholic acid (also known as INT-747), is a potent, orally bioavailable farnesoid X receptor (FXR) agonist. The key role of the farnesoid X receptor (FXR) as a regulator of bile and cholesterol metabolism in the liver, with preclinical data from numerous studies providing strong rationale for the advancement of FXR agonists as hepatoprotective therapeutics in chronic liver disease. Obeticholic acid is marketed under the trade name Ocaliva. Ocaliva is specifically indicated for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in adults unable to tolerate UDCA. — NCATS

Clinical trial activity

40 trials · 22 clinical orgs · 5 marketing orgs

Phase 1

Phase 2

Phase 3

Phase 4

Earliest trial started Jul 1, 2007 (NCT00501592)

Timeline

2000s

Jan 1, 2007
Intercept Pharmaceuticals — Earliest Phase 2 Sponsor(trial)
Apr 9, 2008
Orphan Drug Designation

2010s

Jan 1, 2012
Intercept Pharmaceuticals — Earliest Phase 3 Sponsor(trial)
Jan 1, 2013
Intercept Pharmaceuticals — Earliest Phase 1 Sponsor(trial)
May 27, 2016
Intercept Pharmaceuticals — Earliest FDA Approval
May 27, 2016
Intercept Pharmaceuticals — NDA Secondary Org
May 27, 2016
Intercept Pharmaceuticals — NDA Organization

2020s

May 30, 2023
Apotex — Marketing Organization
May 30, 2023
Lupin — Marketing Organization
May 30, 2023
MSN Laboratories — Marketing Organization
Jul 20, 2023
OPTIMUS PHARMA PRIVATE LTD — Marketing Organization

Mechanism of action

Bile acid receptor FXRAGONIST
NR1H4 agonist
- Bile acid receptor (NR1H4) ↗

Approval history

approvedPriority reviewAcceleratedFast trackMay 27, 2016

Chemistry & pharmacology

Loading structure…

SMILES

CC[C@H]1[C@@H](O)[C@H]2[C@@H]3CC[C@H]([C@H](C)CCC(O)=O)[C@@]3(C)CC[C@@H]2[C@@]4(C)CC[C@@H](O)C[C@@H]14

Mol. weight: 420.6252 g/mol
Lipinski Ro5: 1 violation(s)
Rule of 3: No
Chirality: Single Stereoisomer
Inorganic: No
Polymer: No
Delivery: Oral
Availability: Prescription Only

Oral

Yes

Parenteral

Topical

Black box warningFirst in class

Sources

Also known as

6alpha-ethyl-3alpha,7alpha-dihydroxy-5beta-cholan-24-oic acid
6alpha-ethyl-chenodeoxycholic acid
6alpha-ethyl-chenodeoxycholic acid
6alpha-ethyl-chenodeoxycholic acid
6ecdca
6ecdca
6-ecdca
6-ecdca
6-ecdca
6-ecdca
6-ethyl-3,7-dihydroxycholan-24-oic acid
6-ethyl-cdca
6-ethyl-cdca
6-ethyl chenodeoxycholic acid
6-ethylchenodeoxycholic acid
6-ethylchenodeoxycholic acid
6-ethylchenodeoxycholic acid
6α-ethylchenodeoxycholic acid
6α-ethylchenodeoxycholic acid
cholan-24-oic acid, 6-ethyl-3,7-dihydroxy-, (3alpha,5beta,6alpha,7alpha)-
dsp1747
dsp-1747
dsp-1747
dsp-1747
int747
int 747
int-747
int-747
int-747
int-747
int-747
obeticholic acid
obeticholic acid
obeticholic acid
obeticholic acid
obeticholic acid
obeticholic acid
obeticholic acid
obeticholic acid
obeticholic acid
obeticholic acid
obeticholic acid
ocaliva
ocaliva
ocaliva
ocaliva

Clinical trials

NCT ID	Phase	Start date	Sponsor(s)
NCT06691412	Phase 3	Nov 15, 2024	Assiut University
NCT06488911	Phase 3	Jul 1, 2024	Intercept Pharmaceuticals
NCT06121375	Phase 2/Phase 3 (Phase 3)	Mar 31, 2024	Intercept Pharmaceuticals
NCT07104786	Phase 2	Aug 24, 2023	King Edward Medical University
NCT05223036	Phase 2	Sep 19, 2022	National Cancer Institute (NCI), University of Texas at Houston
NCT05740631	N/A	Aug 22, 2022	Intercept Pharmaceuticals, Katholieke Universiteit Leuven
NCT05112822	Phase 1	Mar 31, 2022	University of Texas at Houston
NCT05239468	Phase 2	Mar 21, 2022	Intercept Pharmaceuticals
NCT04939051	Phase 2	Dec 8, 2021	National Cancer Institute (NCI), University of Michigan
NCT05133830	Phase 1	Nov 23, 2021	GlaxoSmithKline
NCT06715319	Phase 3	Oct 9, 2021	Jinlin University, Shanghai Zhangjiang Biotechnology Limited Company
NCT05450887	Phase 3	Sep 23, 2021	Jiangsu Chia-tai Tianqing Pharmaceutical Co.,Ltd
NCT05573204	Phase 2	Sep 1, 2021	Tanta University
NCT04956328	Phase 3	Jul 22, 2021	Jiangsu Chia-tai Tianqing Pharmaceutical Co.,Ltd
NCT04594694	Phase 2	Oct 2, 2019	Intercept Pharmaceuticals
NCT04053023	Phase 1	Aug 21, 2019	GlaxoSmithKline
NCT03836937	N/A	Mar 5, 2019	Sir Salimullah Medical College
NCT03633227	Phase 4	Jun 22, 2018	Intercept Pharmaceuticals
NCT03439254	Phase 3	Aug 30, 2017	Intercept Pharmaceuticals
NCT02430077	Phase 2	Jun 1, 2016	The University of Texas, Dallas

Showing 20 of 40 trials

Page 1 / 2

Organizations

Research & Development (22)

Organization	Org type	Trials	As lead sponsor	Phases	Earliest year
Intercept Pharmaceuticals	For profit	22	19	5	2007
GlaxoSmithKline	For profit	2	2	1	2019
Indiana University	Academic/Hospital	2	2	2	2014
Jiangsu Chia-tai Tianqing Pharmaceutical Co.,Ltd	For profit	2	2	1	2021
National Cancer Institute (NCI)	Government	2	2	1	2021
National Institute on Alcohol Abuse and Alcoholism (NIAAA)	Government	2	0	2	2014
University of Gothenburg	Academic/Hospital	2	2	2	2013
University of Texas at Houston	Academic/Hospital	2	1	2	2022
Aarhus University	Academic/Hospital	1	1	1	2016
Assiut University	Academic/Hospital	1	1	1	2024
Celerion	For profit	1	0	1	2013
Jinlin University	Academic/Hospital	1	0	1	2021
Katholieke Universiteit Leuven	Academic/Hospital	1	1	1	2022
King Edward Medical University	Academic/Hospital	1	1	1	2023
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Government	1	1	1	2011
Shanghai Zhangjiang Biotechnology Limited Company	For profit	1	1	1	2021
Sir Salimullah Medical College	Academic/Hospital	1	1	1	2019
Tanta University	Academic/Hospital	1	1	1	2021
The University of Texas, Dallas	Academic/Hospital	1	1	1	2016
University of London	Academic/Hospital	1	1	1	2012

22 organizations