FLUDARABINE PHOSPHATE
Fludarabine phosphate treats adults with B-cell chronic lymphocytic leukemia (CLL). It helps patients whose cancer has either progressed or failed to respond after receiving at least one standard treatment involving alkylating agents. This medication is specifically used for these refractory cases, as its benefits for patients who have not yet tried other therapies are not established.
How FLUDARABINE PHOSPHATE Works
This drug works by converting into an active metabolite inside the body's cells. This active form inhibits several enzymes, such as DNA polymerase alpha and ribonucleotide reductase, which are essential for DNA synthesis. By interfering with these processes, the medication prevents the production of new DNA within the cells.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2003-08-28
- Routes
- INJECTION
- Dosage Forms
- INJECTABLE
FLUDARABINE PHOSPHATE Approval History
What FLUDARABINE PHOSPHATE Treats
1 indicationsFLUDARABINE PHOSPHATE is approved for 1 conditions since its original approval in 2003. These indications span multiple therapeutic areas including oncology, immunology, and more.
- B-cell chronic lymphocytic leukemia (CLL) that is refractory or has progressed after standard treatment
FLUDARABINE PHOSPHATE Boxed Warning
SEVERE BONE MARROW SUPPRESSION, CNS TOXICITY, HEMOLYTIC ANEMIA, AND PULMONARY TOXICITY Fludarabine Phosphate Injection should be administered under the supervision of a qualified physician experienced in the use of antineoplastic therapy. Fludarabine phosphate injection can severely suppress bone marrow function. When used at high doses in dose-ranging studies in patients with acute leukemia, fludarabine phosphate was associated with severe neurologic effects, including blindness, coma, and deat...
WARNING: SEVERE BONE MARROW SUPPRESSION, CNS TOXICITY, HEMOLYTIC ANEMIA, AND PULMONARY TOXICITY Fludarabine Phosphate Injection should be administered under the supervision of a qualified physician experienced in the use of antineoplastic therapy. Fludarabine phosphate injection can severely suppress bone marrow function. When used at high doses in dose-ranging studies in patients with acute leukemia, fludarabine phosphate was associated with severe neurologic effects, including blindness, coma, and death. This severe central nervous system toxicity occurred in 36% of patients treated with doses approximately four times greater (96 mg/m 2 /day for 5 to 7 days) than the recommended dose. Similar severe central nervous system toxicity, including coma, seizures, agitation and confusion, has been reported in patients treated at doses in the range of the dose recommended for chronic lymphocytic leukemia [see Warnings and Precautions ( 5.2 )] . Instances of life-threatening and sometimes fatal autoimmune phenomena such as hemolytic anemia, autoimmune thrombocytopenia/thrombocytopenic purpura (ITP), Evans syndrome, and acquired hemophilia have been reported to occur after one or more cycles of treatment with Fludarabine Phosphate Injection. Patients undergoing treatment with Fludarabine Phosphate Injection should be evaluated and closely monitored for hemolysis [see Warnings and Precautions ( 5.3 )]. In a clinical investigation using fludarabine phosphate in combination with pentostatin (deoxycoformycin) for the treatment of refractory chronic lymphocytic leukemia (CLL), there was an unacceptably high incidence of fatal pulmonary toxicity. Therefore, the use of Fludarabine Phosphate Injection in combination with pentostatin is not recommended [see Warnings and Precautions ( 5.5 )]. WARNING: CNS TOXICITY, HEMOLYTIC ANEMIA, AND PULMONARY TOXICITY See full prescribing information for complete boxed warning. Severe central nervous system toxicity occurred in 36% of patients tr
FLUDARABINE PHOSPHATE Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
MoA expansion candidates
Same target(s), different indications — where else is this mechanism being explored?
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
What's emerging in FLUDARABINE PHOSPHATE's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications FLUDARABINE PHOSPHATE treats. First-in-class if their pivotal trials read out positive.
Clinical Trial Registry
890 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT07328503 | 10001986 001986-C | Ph 2 | recruiting | CD22 CAR T-cells to Extend Remission Following Commercial CD19 CAR T-cells in Children, Adolescents, and Adults With Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia |
| NCT05564390 | NCI-2022-07006 NCI-2022-07006, MYELOMATCH | Ph 2 | recruiting | MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial) |
| NCT06615479 | CA088-1007 | Ph 3 | recruiting | A Study to Compare the Efficacy and Safety of BMS-986393 Versus Standard Regimens in Adult Participants With Relapsed or Refractory and Lenalidomide-exposed Multiple Myeloma (QUINTESSENTIAL-2) |
| NCT07583303 | 25704 NCI-2026-02922, 25704 | Ph 1 | not yet recruiting | BSB-2002 After Cyclophosphamide and Fludarabine for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia Patients With NPM1 Mutation |
| NCT06904066 | 10002088 002088-C | Ph 1 | recruiting | Autologous T Cells Transduced With Retroviral Vectors Expressing TCRs for Participant-specific Neoantigens in Patients With Hematologic Malignancies |
| NCT06954987 | NCI-2025-03015 NCI-2025-03015, MM3TCT-A03 | Ph 2 | not yet recruiting | Venetoclax or Placebo in Combination With Reduced-Intensity Conditioning Hematopoietic Cell (Bone Marrow/Blood Stem Cell) Transplant and as Maintenance Therapy After Transplant in Patients With Acute Myeloid Leukemia (A MyeloMATCH Treatment Trial) |
| NCT04539366 | NCI-2020-06646 NCI-2020-06646, PED-CITN-02 | Ph 1 | suspended | Testing a New Immune Cell Therapy, GD2-Targeted Modified T-cells (GD2CART), in Children, Adolescents, and Young Adults With Relapsed/Refractory Osteosarcoma and Neuroblastoma, The GD2-CAR PERSIST Trial |
| NCT07572136 | 10001555 001555-C | Ph 1 | not yet recruiting | Anti-CRLF2-R/TSLPR Chimeric Antigen Receptor T Cells (TSLPR-CART) in Participants With Recurrent or Refractory CRLF2-R/TSLPR-Overexpressing B-Cell Acute Lymphoblastic Leukemia (B-ALL) |
| NCT06698744 | CASE1A24 | Ph 1 | withdrawn | UF-KURE-BCMA CAR-T Cells in Patients With Relapsed or Refractory Multiple Myeloma |
| NCT04093596 UNIVERSAL | ALLO-715-101 | Ph 1 | completed | Safety and Efficacy of ALLO-715 BCMA Allogenic CAR T Cells in in Adults With Relapsed or Refractory Multiple Myeloma (UNIVERSAL) |
| NCT02133196 | 140104 14-C-0104 | Ph 2 | recruiting | T Cell Receptor Immunotherapy for Patients With Metastatic Non-Small Cell Lung Cancer |
| NCT06066359 | 2023-0171 NCI-2023-08318 | Ph 1, Ph 2 | recruiting | Ph I/II Trial of Cord Blood-derived NK Cells With NY-ESO-1 TCR/IL-15 for R/R Myeloma |
| NCT07509034 | 10001973 001973-C | Ph 1 | not yet recruiting | Autologous B7-H3 Chimeric Antigen Receptor T Cells in Previously Treated Extensive-Stage Small Cell Lung Cancer With Recurrent or Refractory Disease |
| NCT07524530 | 10002233 002233-C | Ph 2 | not yet recruiting | Stem Cell Transplantation for Participants With Germline RUNX1 Associated Blood Cancers |
| NCT06897930 | D8313C00001 | Ph 1, Ph 2 | recruiting | A Study to Investigate the Safety, Tolerability, and Efficacy of AZD0120 in Adults With Refractory SLE |
| NCT07479797 | KT-US-740-0603 2025-524403-80-00 | Ph 3 | recruiting | Study Evaluating the Efficacy of KITE-753 Versus Axicabtagene Ciloleucel in Participants With Relapsed or Refractory Large B-Cell Lymphoma After First-Line Therapy |
| NCT07297667 | I246 | Ph 1 | not yet recruiting | GCAR1, a Chimeric Antigen Receptor (CAR) T-CELL Therapy for Relapsed/Refractory GPNMB-Expressing Solid Tumours |
| NCT07526493 | QH10310-NADs-01（0） | Ph 1 | recruiting | Safety and Pharmacodynamics of QH103 Cell Injection in the Treatment of Patients With Relapsed/Refractory Antibody-Mediated Neurological Autoimmune Diseases. |
| NCT05088356 | IRB-60439 NCI-2021-12228 | Ph 1 | active not recruiting | Reduced Intensity Allogeneic HCT in Advanced Hematologic Malignancies w/T-Cell Depleted Graft |
| NCT07582172 | 25663 NCI-2026-03208, 25663 | Ph 2 | not yet recruiting | Total Marrow and Lymphoid Irradiation in Combination With Fludarabine and Melphalan as Conditioning for Allogeneic Peripheral Blood Stem Cell Hematopoietic Cell Transplant in Older Patients With Refractory and Relapsed Acute Myeloid Leukemia and High-risk Myelodysplastic Syndrome |
| NCT05020444 | 20-518 | Ph 1 | recruiting | TriPRIL CAR T Cells in Multiple Myeloma |
| NCT07257419 | HAPALL NCI-2025-08364 | Ph 1 | recruiting | CD45RA-depleted CD19-CAR T Cell Consolidation After TCRαβ+/CD19 B Cell-depleted Haploidentical Hematopoietic Cell Transplantation for Relapsed/Refractory CD19+ ALL and Lymphoma |
| NCT06996119 | 23822 NCI-2025-03603, 23822 | Ph 1 | recruiting | Emapalumab With Post-Transplant Cyclophosphamide, Tacrolimus and Mycophenolate Mofetil for the Prevention of Graft-versus-Host Disease After Donor Reduced-Intensity Hematopoietic Cell Transplant |
| NCT04923893 CARTITUDE-5 | CR109015 68284528MMY3004, 2021-001242-35 | Ph 3 | active not recruiting | A Study of Bortezomib, Lenalidomide and Dexamethasone (VRd) Followed by Cilta-cel, a CAR-T Therapy Directed Against BCMA Versus VRd Followed by Lenalidomide and Dexamethasone (Rd) Therapy in Participants With Newly Diagnosed Multiple Myeloma for Whom ASCT is Not Planned as Initial Therapy |
| NCT06253520 | 10001662 001662-C | Ph 1 | active not recruiting | Autologous T-cells Genetically Engineered to Express Receptors Reactive Against KRAS Mutations in Conjunction With a Vaccine Directed Against These Antigens in Participants With Metastatic Cancer |
| NCT01174121 | 100166 10-C-0166 | Ph 2 | recruiting | Immunotherapy Using Tumor Infiltrating Lymphocytes for Patients With Metastatic Cancer |
| NCT07575893 REACH-CD30 | NYMC 628 | Ph 1 | not yet recruiting | CD30 CAR-T Cells for Low Risk Relapsed Classical Hodgkin Lymphoma |
| NCT03412877 | 180049 18-C-0049 | Ph 2 | recruiting | Administration of Autologous T-Cells Genetically Engineered to Express T-Cell Receptors Reactive Against Neoantigens in People With Metastatic Cancer |
| NCT02621021 | 160027 16-C-0027 | Ph 2 | recruiting | A Phase 2 Trial for Metastatic Melanoma Using Adoptive Cell Therapy With Tumor Infiltrating Lymphocytes Plus IL-2 Either Alone or Following the Administration of Pembrolizumab |
| NCT06577025 aMMbition | 54767414MMY2093 54767414MMY2093, 2023-505792-71-00 | Ph 2 | active not recruiting | A Study of Different Sequences of Cilta-cel, Talquetamab in Combination With Daratumumab and Teclistamab in Combination With Daratumumab Following Induction With Daratumumab, Bortezomib, Lenalidomide and Dexamethasone in Participants With Standard-risk Newly Diagnosed Multiple Myeloma |
| NCT07444632 | 2025-1398 NCI-2026-01395 | Ph 1 | not yet recruiting | Phase1 Basket Trial Of CAR.70-Engineered IL15-Transduced With TGFBR2 Knock Out Cord Blood-Derived NK Cells For Relapsed/Refractory Lymphoid Malignancies |
| NCT07015983 | CA061-1011 2024-519278-37 | Ph 2 | recruiting | A Study of CC-97540 (BMS-986353 or Zola-cel), CD19-Targeted NEX-T CAR T Cells, in Participants With Active SLE Despite Immunosuppressants (Breakfree-SLE) |
| NCT05027945 | 10000404 000404-C | Ph 2 | recruiting | A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome |
| NCT07149857 CARTITUDE-10 | 68284528MMY2012 68284528MMY2012, 2025-521975-30-00 | Ph 2 | recruiting | A Study to Evaluate Efficacy and Safety of Ciltacabtagene Autoleucel |
| NCT06481735 | CHN-PLAGH-BT-087 | Ph 1, Ph 2 | recruiting | TCR Reserved and Power3 (SPPL3) Gene Knock-out Allogeneic CD19-targeting CAR-T Cell Therapy in r/r B-ALL |
| NCT07428486 | 2025-1676 NCI-2026-01274 | Ph 1 | withdrawn | A Phase 1 Study Of FLAG Chemotherapy In Combination With Lisaftoclax And Pelcitoclax In Patients With Relapsed/Refractory T-Cell Acute Lymphoblastic Leukemia |
| NCT06034470 | RG1123378 NCI-2023-03572, FHIRB0020122 | Ph 1 | active not recruiting | Combination Chemotherapy (FLAG-Ida) With Pivekimab Sunirine (PVEK) for the Treatment of Newly Diagnosed Adverse Risk Acute Myeloid Leukemia and Other High-Grade Myeloid Neoplasms |
| NCT05826535 | LYL314-101 | Ph 1, Ph 2 | recruiting | Study of Rondecabtagene Autoleucel in Aggressive Large B-Cell Lymphoma |
| NCT06413498 iMMagine-3 | KT-US-679-0788 2024-511188-26, jRCT2043240170 | Ph 3 | recruiting | A Study Comparing Anitocabtagene Autoleucel to Standard of Care Therapy in Participants With Relapsed/ Refractory Multiple Myeloma |
| NCT06777979 | 1922CAR NCI-2024-10103 | Ph 1 | recruiting | CD19-CD22-Bispecific Chimeric Antigen Receptor (CAR) T Cell Therapy for Pediatric Patients With Acute Lymphoblastic Leukemia |
| NCT07573332 | CA061-1031 | Ph 1 | not yet recruiting | A Safety and Tolerability Study of CC- 97540 (BMS-086353) in Anti-Aquaporin 4 Antibody Positive Neuromyelitis Optica Patients |
| NCT05922930 | 2022-0687 NCI-2023-05027 | Ph 1, Ph 2 | recruiting | Study of TROP2 CAR Engineered IL15-transduced Cord Blood-derived NK Cells Delivered Intraperitoneally for the Management of Platinum Resistant Ovarian Cancer, Mesonephric-like Adenocarcinoma, and Pancreatic Cancer |
| NCT06043323 | 2023-0087 NCI-2023-07173 | Ph 2 | recruiting | A Phase II Study of Axicabtagene Ciloleucel, an Anti-CD19 Chimeric Antigen Receptor (CAR) Tcell Therapy, in Combination With Radiotherapy (RT) in Relapsed/Refractory Follicular Lymphoma |
| NCT07164469 | 2025-0986 NCI-2025-06569 | Ph 2 | not yet recruiting | Phase 2 Trial of CD70.CAR NK Cells for Patients With Primary Refractory or Early Relapsed Diffuse Large B-Cell Lymphoma and Hodgkin Lymphoma |
| NCT06500273 ALPHA3 | ALLO-501A-202 | Ph 2 | recruiting | Consolidation of First-Line MRD+ Remission With Cema-cel in Patients With LBCL |
| NCT05092451 | 2021-0386 NCI-2021-12093 | Ph 1, Ph 2 | recruiting | Phase I/II Study of CAR.70- Engineered IL15-transduced Cord Blood-derived NK Cells in Conjunction With Lymphodepleting Chemotherapy for the Management of Relapse/Refractory Hematological Malignances |
| NCT07569965 FRONTIER | PROJ112909 U24HL138660 | Ph 2 | not yet recruiting | Frontline Risk-Adapted Optimization of Novel Targeted Immunotherapy Evaluation in High-Risk MCL |
| NCT04872595 | 21-193 | Ph 2 | active not recruiting | A Modified Dose of Rabbit Anti-thymocyte Globulin (rATG) in Children and Adults Receiving Treatment to Help Prepare Their Bodies for a Bone Marrow Transplant |
| NCT06930651 | 2024-1967 NCI-2025-02697 | Ph 1, Ph 2 | recruiting | A Phase I/II Study of CAR.70-Engineered IL15-Transduced Cord Blood-Derived NK Cells With TGF-beta Receptor 2 (TGFBR2) Knock Out in Conjunction With Lymphodepleting Chemotherapy for the Management of Relapsed/Refractory Myeloid Malignancies |
| NCT07101432 | 2025-0561 NCI-2025-05464 | Ph 1 | recruiting | Phase I Study of Preconditioning Radiation Therapy With IL-15 Transduced TGFBR2 KO CAR.TROP2-engineered Cord Blood-derived NK Cells in Patients With Advanced Head and Neck Cancer (RADIANCE-NK) |
Showing 50 of 890 trials
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
FLUDARABINE PHOSPHATE FDA Label Details
Indications & Usage
FDA Label (PDF)FLUDARABINE PHOSPHATE is indicated for the treatment of B-cell chronic lymphocytic leukemia (CLL) that is refractory or has progressed after standard treatment.
WARNING: SEVERE BONE MARROW SUPPRESSION, CNS TOXICITY, HEMOLYTIC ANEMIA, AND PULMONARY TOXICITY Fludarabine Phosphate Injection should be administered under the supervision of a qualified physician experienced in the use of antineoplastic therapy. Fludarabine phosphate injection can severely suppres...
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FLUDARA
Full clinical data, patents, trials, and competitive landscape for fludarabine phosphate.
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment