pentamidine

Trade name: pentam

Small moleculeapprovedOrphan Drug ^FDA

Approved

Oct 16, 1984

Pentamidine (formulated as a salt, pentamidine diisethionate or dimesilate) is an antimicrobial medication given for prevention and treatment of pneumocystis pneumonia (PCP) caused by Pneumocystis jirovecii (formerly known as Pneumocystis carinii), a severe interstitial type of pneumonia often seen in patients with HIV infection. The drug is also the mainstay of treatment for stage I infection with Trypanosoma bruceigambiense (West African trypanosomiasis). Pentamidine is also used as a prophylactic against PCP in patients receiving chemotherapy and in some patients who have undergone organ transplantation, as they also have a depressed immune system as a direct side-effect of the drugs used. The mortality of untreated PCP is very high. Additionally, pentamidine has good clinical activity in treating leishmaniasis, and yeast infections caused by the organism Candida albicans. Pentamidine is also used as a prophylactic antibiotic for children undergoing treatment for leukemia. Studies suggest that the pentamidine isethionate interferes with microbial nuclear metabolism by inhibition of DNA, RNA, phospholipid and protein synthesis. However, the mode of action is not fully understood. — NCATS

Clinical trial activity

36 trials · 38 clinical orgs · 12 marketing orgs

Phase 1

Phase 2

Phase 3

Phase 4

Earliest trial started Jun 1, 1993 (NCT00002524)

Timeline

1980s

Feb 28, 1984
Orphan Drug Designation
Oct 16, 1984
Fresenius — Earliest FDA Approval
Oct 16, 1984
Fresenius — NDA Secondary Org
Oct 16, 1984
Fresenius — NDA Organization

1990s

Jun 30, 1992
Hospira — Marketing Organization
Jan 1, 1993
Earliest Phase 2 Sponsor(trial)
Aug 17, 1995
Watson Pharmaceuticals — Marketing Organization
Dec 18, 1995
Baxalta — Marketing Organization
Dec 18, 1995
Baxter — Marketing Organization
Jan 1, 1999
Earliest Phase 1 Sponsor(trial)
Jan 1, 1999
Earliest Phase 3 Sponsor(trial)

2010s

Sep 28, 2017
Seton Pharm — Marketing Organization
Sep 28, 2017
Pharmaceutical Associates — Marketing Organization

2020s

Jan 7, 2021
Emcure — Marketing Organization
Jan 7, 2021
Avet Lifesciences — Marketing Organization
Mar 17, 2022
X Gen Pharmaceuticals — Marketing Organization
Mar 17, 2022
PHARMOBEDIENT — Marketing Organization

Mechanism of action

Kinetoplast DNAINHIBITOR
Kinetoplast DNA inhibitor
Binds DNA, concentrated in kinetoplast
DNAINHIBITOR
DNA inhibitor
Binds minor groove of DNA

Approval history

approvedPriority reviewOct 16, 1984

Chemistry & pharmacology

Loading structure…

SMILES

N=C(N)c1ccc(OCCCCCOc2ccc(C(=N)N)cc2)cc1

Mol. weight: 340.43 g/mol
Lipinski Ro5: Pass
Rule of 3: No
Chirality: Achiral Molecule
Inorganic: No
Polymer: No
Delivery: Parenteral, Topical
Availability: Prescription Only

Oral

Parenteral

Yes

Topical

Yes

Sources

Also known as

1,5-bis(4-amidinophenoxy)pentane
1,5-bis(4-amidinophenoxy)pentane
4,4'-(1,5-pentanediylbis(oxy))bis-benzenecarboximidamide
4,4'-(1,5-pentanediylbis(oxy))bis-benzenecarboximidamide
4,4'-diamidinodiphenoxypentane
4,4'-diamidinodiphenoxypentane
4,4'-(pentamethylenedioxy)dibenzamidine
4,4'-(pentamethylenedioxy)dibenzamidine
gnf-pf-3680
gnf-pf-3680
mb-800
mb-800
mb-800 [as isethionate]
mb-800 [as isethionate]
nebupent
nebupent
nebupent [as isethionate]
nebupent [as isethionate]
pentacarinat
pentacarinat
pentacarinat [as isethionate]
pentacarinat [as isethionate]
pentam
pentam
pentam
pentam 300 [as isethionate]
pentam 300 [as isethionate]
pentamidin
pentamidin
pentamidin
pentamidina
pentamidina
pentamidindiisetionat
pentamidindiisetionat
pentamidine
pentamidine
pentamidine
pentamidine
pentamidine
pentamidine
pentamidine
pentamidine
pentamidine diisethionate
pentamidine diisethionate
pentamidine diisethionate
pentamidine isethionate
pentamidine isethionate
pentamidine isethionate
pentamidine isethionate
pentamidine isethionate
pentamidine isetionate
pentamidine isetionate
pentamidine isetionate
pentamidine isetionate
pentamidine isetionate
pentamidine isetionate
pentamidine isetionate
pentamidine mesilate
pentamidine mesylate
pentamidinum
pentamidinum
p,p'-(pentamethylenedioxy)dibenzamidine
p,p'-(pentamethylenedioxy)dibenzamidine
rp-2512
rp-2512
rp-2512 [as isethionate]
rp-2512 [as isethionate]

Clinical trials

NCT ID	Phase	Start date	Sponsor(s)
NCT03730363	Phase 1	Dec 19, 2018	University of Kentucky
NCT03403621	Phase 1	Mar 5, 2018	Mayo Clinic
NCT03201159	Phase 1	Jun 25, 2017	National Institute on Alcohol Abuse and Alcoholism (NIAAA), The Cleveland Clinic, The University of Texas, Dallas, University of Louisville, University of Massachusetts
NCT03096457	Phase 2/Phase 3 (Phase 3)	Apr 15, 2017	Fundacion Nacional de Dermatologia
NCT03445897	Phase 2	Jan 31, 2016	AB Foundation for Medical Research
NCT02210182	Phase 1	Aug 1, 2014	Verlyx Pharma Inc
NCT02919605	Phase 2	Jan 1, 2014	Fundação de Medicina Tropical Dr. Heitor Vieira Dourado
NCT01360762	Phase 3	Nov 1, 2011	Addis Ababa University, Amhara Regional Health Bureau, Amhara Region, Drugs for Neglected Diseases Initiative, Institute of Tropical Medicine, Belgium, Leishmania East Africa Platform, Medecins Sans Frontieres, Tigray Regional Health Bureau, Tigray Region, University of Antwerp, University of Gondar
NCT00810953	Phase 1/Phase 2 (Phase 2)	Jan 1, 2009	McGill University, Verlyx Pharma Inc
NCT00729807	Phase 2	Jul 1, 2008	National Cancer Institute (NCI), University of Maryland, University of Maryland, Baltimore County
NCT00809796	Phase 1/Phase 2 (Phase 2)	Mar 1, 2008	McGill University, Verlyx Pharma Inc
NCT00636935	Phase 4	Feb 1, 2008	George Washington University
NCT00078559	Phase 1/Phase 2 (Phase 2)	Nov 1, 2003	Immune Tolerance Network, University of Wisconsin
NCT00803933	Phase 2	Feb 1, 2003	Bill and Melinda Gates Foundation, Immtech Pharmaceuticals, Inc, Programme Nationale de Lutte contre la Trypanosomiase Humaine Africaine
NCT00014911	Phase 2	Apr 1, 2001	Immune Tolerance Network, National Institute of Allergy and Infectious Diseases (NIAID), University of Alberta
NCT00002055	N/A	Nov 2, 1999	Fisons
NCT00002056	N/A	Nov 2, 1999	Lyphomed, Inc.
NCT00002291	N/A	Nov 2, 1999	Fisons
NCT00000707	N/A	Nov 2, 1999	National Institute of Allergy and Infectious Diseases (NIAID)
NCT00002340	Phase 3	Nov 2, 1999	Glaxo Wellcome

Showing 20 of 36 trials

Page 1 / 2

Organizations

Research & Development (38)

Organization	Org type	Trials	As lead sponsor	Phases	Earliest year
National Institute of Allergy and Infectious Diseases (NIAID)	Government	12	12	4	1999
Fisons	For profit	5	5	1	1999
Verlyx Pharma Inc	For profit	3	3	2	2008
Fujisawa	For profit	2	0	2	1999
Glaxo Wellcome	For profit	2	2	2	1999
Immune Tolerance Network	Academic/Hospital	2	0	1	2001
Lyphomed, Inc.	For profit	2	2	1	1999
McGill University	Academic/Hospital	2	0	1	2008
National Cancer Institute (NCI)	Government	2	0	1	1993
AB Foundation for Medical Research	Academic/Hospital	1	1	1	2016
Addis Ababa University	Academic/Hospital	1	0	1	2011
Amhara Regional Health Bureau, Amhara Region	Government	1	0	1	2011
Bill and Melinda Gates Foundation	Academic/Hospital	1	0	1	2003
Drugs for Neglected Diseases Initiative	Academic/Hospital	1	0	1	2011
Fundação de Medicina Tropical Dr. Heitor Vieira Dourado	Academic/Hospital	1	1	1	2014
Fundacion Nacional de Dermatologia	Academic/Hospital	1	1	1	2017
George Washington University	Academic/Hospital	1	1	1	2008
Immtech Pharmaceuticals, Inc	For profit	1	1	1	2003
Institute of Tropical Medicine, Belgium	Academic/Hospital	1	1	1	2011
Jacobus Pharmaceutical	For profit	1	0	1	1999

38 organizations