Last reviewed 2026-04-20 · How we verify

Fludara (FLUDARABINE PHOSPHATE)

Fludara (generic name: FLUDARABINE PHOSPHATE) is a Nucleoside Metabolic Inhibitor Small molecule drug developed by Nihon Schering K.K.. It is currently FDA-approved (first approved 1991) for Acute myeloid leukemia, disease, Chronic lymphoid leukemia, disease, Low-grade B cell non-Hodgkin lymphoma.

Fludara works by interfering with the production of DNA and RNA in cancer cells, ultimately leading to cell death.

Fludara (Fludarabine Phosphate) is a nucleoside metabolic inhibitor, a small molecule drug that targets carbonic anhydrase 5A, mitochondrial. It was originally developed by Nihon Schering K.K. and is now owned by Genzyme Corp. Fludara is approved to treat various types of leukemia and lymphoma, including acute myeloid leukemia, chronic lymphoid leukemia, low-grade B cell non-Hodgkin lymphoma, and mantle cell lymphoma. The drug is off-patent and has multiple generic manufacturers. Key safety considerations include potential bone marrow suppression and increased risk of infections.

At a glance

Mechanism of action

Fludarabine phosphate is rapidly dephosphorylated to 2-fluoro-ara-A and then phosphorylated intracellularly by deoxycytidine kinase to the active triphosphate, 2-fluoro-ara-ATP. This metabolite appears to act by inhibiting DNA polymerase alpha, ribonucleotide reductase and DNA primase, thus inhibiting DNA synthesis. The mechanism of action of this antimetabolite is not completely characterized and may be multi-faceted.

Approved indications

• Acute myeloid leukemia, disease
• Chronic lymphoid leukemia, disease
• Low-grade B cell non-Hodgkin lymphoma
• Mantle cell lymphoma

Boxed warnings

• 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 treated with doses approximately four times greater (96 mg/m 2 /day for 5 to 7 days) than the recommended dose. This toxicity was seen in ≤0.2% of patients treated at the recommended dose levels (25 mg/m 2 ). ( 5.1 ) Instances of life-threatening and sometimes fatal autoimmune hemolytic anemia have been reported after one or more cycles of treatment. ( 5.3 ) In a clinical investigation of the combination of fludarabine phosphate with pentostatin (deoxycoformycin) for the treatment of refractory chronic lymphocytic leukemia (CLL), there was an unacceptably high incidence of fatal pulmonary toxicity. ( 5.5 )

Common side effects

• Myelosuppression
• Neutropenia
• Thrombocytopenia
• Anemia
• Pneumonia
• Infection
• Fever
• Chills
• Fatigue
• Weakness
• Malaise
• Mucositis

Key clinical trials

• Metabolically Fit CD19 CAR T-cell Therapy With CD34 Selection in Patients With CD19+ Relapsed/Refractory NHL, CLL/SLL (PHASE1,PHASE2)
• A Study Comparing Anitocabtagene Autoleucel to Standard of Care Therapy in Participants With Relapsed/ Refractory Multiple Myeloma (PHASE3)
• RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy (PHASE2,PHASE3)
• Pediatric-Inspired Regimen Combined With Venetoclax and Immunotherapy for Adult Ph-Negative Acute Lymphoblastic Leukemia (NA)
• Administering Peripheral Blood Lymphocytes Transduced With a CD70-Binding Chimeric Antigen Receptor to People With CD70 Expressing Cancers (PHASE1,PHASE2)
• 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) (PHASE2)
• MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial) (PHASE2)
• Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant of DOCK8 Deficiency (PHASE2)

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• Fludara CI brief — competitive landscape report
• Fludara updates RSS · CI watch RSS
• Nihon Schering K.K. portfolio CI

Frequently asked questions about Fludara

Related

• Drug class: All Nucleoside Metabolic Inhibitor drugs
• Target: All drugs targeting Carbonic anhydrase 5A, mitochondrial
• Manufacturer: Nihon Schering K.K. — full pipeline
• Therapeutic area: All drugs in Oncology
• Indication: Drugs for Acute myeloid leukemia, disease
• Indication: Drugs for Chronic lymphoid leukemia, disease
• Indication: Drugs for Low-grade B cell non-Hodgkin lymphoma

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing