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cladribine

Trade name: leustatin

Small moleculeapprovedOrphan Drug FDA

Approved

Feb 26, 1993

Cladribine is used for the treatment of hairy cell leukemia and multiple sclerosis (MS). As a purine analog, it is a synthetic anti-cancer agent that also suppresses the immune system. Chemically, it mimics the nucleoside adenosine and thus inhibits the enzyme adenosine deaminase, which interferes with the cell's ability to process DNA. It can be distinguished from other chemotherapeutic agents affecting purine metabolism in that it is cytotoxic to both actively dividing and quiescent lymphocytes and monocytes, inhibiting both DNA synthesis and repair. Cladribine injection is a potent antineoplastic agent with potentially significant toxic side effects. In MS, the novel mechanism of action of cladribine is expected to reduce inflammation, autoimmune effects and autoreactive cell damage, thereby improving the integrity of the blood–brain barrier. Thus, the effects of cladribine may target some of the key events that are central to the pathophysiology of MS. — NCATS

Clinical trial activity

125 trials · 140 clinical orgs · 10 marketing orgs

Phase 1
32
Phase 2
81
Phase 3
23
Phase 4
12

Earliest trial started Oct 1, 1994 (NCT00002833)

Timeline

1990s

  1. Nov 15, 1990

    Orphan Drug Designation

  2. Feb 26, 1993

    Johnson and Johnson — Earliest FDA Approval

  3. Feb 26, 1993

    Johnson and Johnson — NDA Secondary Org

  4. Feb 26, 1993

    Johnson and Johnson — NDA Organization

  5. Jan 1, 1994

    Earliest Phase 2 Sponsor(trial)

  6. Jan 1, 1995

    Earliest Phase 1 Sponsor(trial)

  7. Jan 1, 1998

    Earliest Phase 3 Sponsor(trial)

2000s

  1. Feb 28, 2000

    West-Ward Pharmaceutical — Marketing Organization

  2. Feb 28, 2000

    Hikma — Marketing Organization

  3. Apr 22, 2004

    Fresenius — Marketing Organization

2010s

  1. Oct 6, 2011

    Mylan — Marketing Organization

  2. Oct 6, 2011

    PHARMOBEDIENT — Marketing Organization

  3. Mar 29, 2019

    Merck Serono — NDA Secondary Org

  4. Nov 29, 2019

    Zhejiang Hisun Pharmaceutical Co. Ltd. — Marketing Organization

2020s

  1. Nov 24, 2025

    Apotex — Marketing Organization

  2. Mar 27, 2026

    HOPEWELL PHARMA — Marketing Organization

Indications

Approved for

Studied for

Mechanism of action

  • DNAINHIBITOR

    DNA inhibitor

    Activated by the deoxycytidine kinase to a toxic compound, which then gets incorporated into DNA

Approval history

  • approvedFeb 26, 1993

Chemistry & pharmacology

Loading structure…

SMILES

NC1=NC(Cl)=NC2=C1N=CN2[C@H]3C[C@H](O)[C@@H](CO)O3
Mol. weight
285.687 g/mol
Lipinski Ro5
Pass
Rule of 3
No
Chirality
Single Stereoisomer
Inorganic
No
Polymer
No
Delivery
Oral, Parenteral
Availability
Prescription Only

Oral

Yes

Parenteral

Yes

Topical

No

Black box warningProdrug

Sources

Also known as

  • 2cda
  • 2cda
  • 2-cda
  • 2-cda
  • 2-cda
  • 2-chloro-2'-deoxyadenosine
  • 2-chloro-2'-deoxyadenosine
  • 2-chloro-2'-deoxy-beta-adenosine
  • 2-chloro-2'-deoxy-beta-adenosine
  • 2-chloro-6-amino-9-(2-deoxy-beta-d-erythro-pentofuranosyl)purine
  • 2-chloro-6-amino-9-(2-deoxy-beta-d-erythro-pentofuranosyl)purine
  • 2-chlorodeoxyadenosine
  • 2-chlorodeoxyadenosine
  • 2-chlorodeoxyadenosine
  • 2-chloro-deoxyadenosine
  • 2-chloro-deoxyadenosine
  • 2clado
  • 2clado
  • (2r,3s,5r)-5-(6-amino-2-chloropurin-9-yl)-2-(hydroxymethyl)oxolan-3-ol
  • (2r,3s,5r)-5-(6-amino-2-chloropurin-9-yl)-2-(hydroxymethyl)oxolan-3-ol
  • cda
  • cladarabine
  • cladaribine
  • cladaribine
  • cladribina
  • cladribina
  • cladribina
  • cladribine
  • cladribine
  • cladribine
  • cladribine
  • cladribine
  • cladribine
  • cladribine
  • cladribine
  • cladribine
  • cladribine
  • cladribinum
  • cladribinum
  • cldado
  • cldado
  • leustat
  • leustat
  • leustat
  • leustatin
  • leustatin
  • leustatin
  • leustatin
  • leustatin
  • leustatine
  • litak
  • litak
  • litak
  • litak
  • litak
  • litak
  • rwj-26251
  • rwj-26251
  • rwj-26251

Clinical trials

NCT IDPhaseStart dateSponsor(s)
NCT06965114Phase 1/Phase 2 (Phase 2)Jun 27, 2026National Cancer Institute (NCI), Ohio State University
NCT07311746Phase 1/Phase 2 (Phase 2)Jun 1, 2026University of Texas at Houston
NCT07053020Phase 1/Phase 2 (Phase 2)Dec 1, 2025University of Texas at Houston
NCT07032727Phase 2Sep 12, 2025Rigel Pharmaceuticals, University of Texas at Houston
NCT06763666Phase 4Feb 28, 2025Central People's Hospital of Zhanjiang, China Medical University, First People's Hospital of Foshan, Guangdong Medical University, Guangdong Second Provincial Traditional Chinese Medicine Hospital, Guangzhou Medical University, Guangzhou Panyu Central Hospital, Guilin University, Hacettepe University, Hainan People's Hospital, Huizhou People's Central Hospital, Jiangmen Central Hospital, Maoming People's Hospital, Peking University, People's Liberation Army of China, Shantou University, Southern Medical University, Sun Yat-Sen University, Xiamen University, Yuebei People's Hospital
NCT06561152Phase 1/Phase 2 (Phase 2)Jan 31, 2025Pipeline Therapeutics, Inc., Stanford University
NCT06474663Phase 1Dec 31, 2024University of Texas at Houston
NCT06660368Phase 2Nov 30, 2024AbbVie, University of South Florida
NCT06504459Phase 2Oct 31, 2024AbbVie, OHSU Knight Cancer Institute, Oregon Health and Science University
NCT06561360Phase 2Sep 9, 2024Genentech, Memorial Sloan-Kettering Cancer Center
NCT06532552Phase 2Jul 29, 2024Anhui Medical University, Bengbu Medical College, Huazhong University of Science and Technology, Nanjing Medical University, Qingdao University, Shandong University, Soochow University
NCT06463587Phase 3Jun 25, 2024Merck KGaA, Merck Serono
NCT05766514Phase 2Mar 31, 2024University of Florida
NCT05780879Phase 2Feb 29, 2024Genentech, University of Michigan
NCT06007911Phase 1Feb 29, 2024Medical College of Wisconsin
NCT06232655Phase 2Feb 8, 2024The Leukemia and Lymphoma Society, University of Colorado, Denver
NCT06133556Phase 2Nov 24, 2023China Academy of Chinese Medical Sciences
NCT06021600Phase 1Oct 9, 2023University of Texas at Houston
NCT06060782Phase 1Oct 1, 2023Sun Yat-Sen University
NCT05906914Phase 2Jun 29, 2023Zhejiang University
Showing 20 of 125 trials
Page 1 / 7

Organizations

Research & Development (140)

OrganizationOrg typeTrialsAs lead sponsorPhasesEarliest year
National Cancer Institute (NCI)Government23841994
University of Texas at HoustonAcademic/Hospital181831994
Merck SeronoFor profit10542005
University of WashingtonAcademic/Hospital101032014
Merck KGaAFor profit8732008
Sun Yat-Sen UniversityAcademic/Hospital6542016
Jiao Tong UniversityAcademic/Hospital5522016
Soochow UniversityAcademic/Hospital5532014
People's Liberation Army of ChinaGovernment4022016
AbbVieFor profit3022021
Bengbu Medical CollegeAcademic/Hospital3022022
Central South UniversityAcademic/Hospital3012016
Fujian Medical UniversityAcademic/Hospital3012016
GenentechFor profit3012004
Huazhong University of Science and TechnologyAcademic/Hospital3022016
J. W. Goethe UniversityAcademic/Hospital3322003
Mayo ClinicAcademic/Hospital3021996
Medical College of WisconsinAcademic/Hospital3322017
Nanjing Medical UniversityAcademic/Hospital3022022
Sichuan UniversityAcademic/Hospital3322017
140 organizations
Page 1 / 7

Marketing (11)

OrganizationOrg typeRelationshipDate
ApotexFor profitMKTGNov 24, 2025
FreseniusFor profitMKTGApr 22, 2004
HOPEWELL PHARMAFor profitMKTGMar 27, 2026
HikmaFor profitMKTGFeb 28, 2000
Johnson and JohnsonFor profitNDA2Feb 26, 1993
Johnson and JohnsonFor profitNDAFeb 26, 1993
Merck SeronoFor profitNDA2Mar 29, 2019
MylanFor profitMKTGOct 6, 2011
PHARMOBEDIENTFor profitMKTGOct 6, 2011
West-Ward PharmaceuticalFor profitMKTGFeb 28, 2000
Zhejiang Hisun Pharmaceutical Co. Ltd.For profitMKTGNov 29, 2019