Nelarabine

Nelarabine is a prodrug of the deoxyguanosine analogue 9-β- D -arabinofuranosylguanine (ara-G), a nucleoside metabolic inhibitor. Nelarabine is demethylated by adenosine deaminase (ADA) to ara-G, mono-phosphorylated by deoxyguanosine kinase and deoxycytidine kinase, and subsequently converted to the active 5’-triphosphate, ara-GTP.

• Nelarabine injection is indicated for the treatment of T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL) in adult and pediatric patients age 1 year and older whose disease has not responded to or has relapsed following treatment with at least two chemotherapy regimens. Nelarabine injection is a nucleoside metabolic inhibitor indicated for the treatment of patients with T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma in adult and pediatric patients age 1 year and older whose disease has not responded to or has relapsed following treatment with at least two chemotherapy regimens.ICD-10: C85.90, C95.90

• None.contraindicated

Adult Dose : 1,500 mg/m² administered intravenously over 2 hours on Days 1, 3, and 5 repeated every 21 days. ( 2.1 ) Pediatric Dose : 650 mg/m² administered intravenously over 1 hour daily for 5 consecutive days repeated every 21 days. ( 2.1 ) Discontinue treatment for neurologic reactions greater than or equal to Grade 2. ( 2.2 ) Dosage may be delayed for hematologic reactions. ( 2.2 ) Take measures to prevent hyperuricemia. ( 2.4 ) 2.1 Recommended Dosage This product is for intravenous use only. Adult Dosage : The recommended adult dose of nelarabine injection is 1,500 mg/m² administered intravenously over 2 hours on Days 1, 3, and 5 repeated every 21 days. Administer nelarabine injection undiluted. Pediatric Dosage : The recommended pediatric dose of nelarabine injection is 650 mg/m² administered intravenously over 1 hour daily for 5 consecutive days repeated every 21 days. Administer nelarabine injection undiluted. The recommended duration of treatment for adult and pediatric patients has not been clearly established. In clinical trials, treatment was generally continued until there was evidence of disease progression, the patient experienced unacceptable toxicity, the patient became a candidate for hematopoietic stem cell transplantation (HSCT), or the patient no longer continued to benefit from treatment. 2.2 Dosage Modification Discontinue nelarabine injection if the patient develops a neurologic adverse reaction of NCI CTCAE Grade 2 or greater.

Neurologic Adverse Reactions : Severe neurologic reactions have been reported. Monitor for signs and symptoms of neurologic toxicity. ( 5.1 ) Hematologic Reactions : Complete blood counts including platelets should be monitored regularly. ( 5.2 ) Embryo-Fetal Toxicity : Can cause fetal harm. Advise females of reproductive potential of the potential risk to the fetus and to use effective contraception; and advise males to use condoms. ( 5.3 , 8.1 , 8.3 ) Effects on Ability to Drive and Use Machines : Somnolence may occur. Advise patients to refrain from these activities until somnolence has resolved. ( 5.6 ) 5.1 Neurologic Adverse Reactions Nervous system adverse reactions of any grade were reported for 223 (76%) adult patients across the Phase I and Phase II trials, and Grade 3 or higher (severe, life-threatening, or fatal) adverse reactions were reported for 55 (19%) patients following initiation of nelarabine injection therapy [see Adverse Reactions ( 6.1 )] . Based on patients with complete data, the median time to onset of first event is 5 days from start of first infusion (range: 1-166), and the median duration is 6 days (range: 1-393 days). Nervous system adverse reactions of any grade were reported for 69 (42%) pediatric patients across the Phase I and Phase II trials, and Grade 3 or higher (severe, life-threatening, or fatal) adverse reactions were reported for 25 (15%) patients following initiation of nelarabine injection therapy [see Adverse Reactions ( 6.1 )] .

The following clinically-significant adverse reactions are discussed in greater detail in other sections of the label: Neurologic [see Boxed Warning, Warnings and Precautions ( 5.1 )] Hematologic [see Warnings and Precautions ( 5.2 )] Tumor Lysis Syndrome [see Warnings and Precautions ( 5.4 )] Effects on Ability to Drive and Use Machines [see Warnings and Precautions ( 5.6 )] The most common (≥ 20%) adverse reactions were: Adult: anemia, thrombocytopenia, neutropenia, nausea, diarrhea, vomiting, constipation, fatigue, pyrexia, cough, and dyspnea ( 6.1 ) Pediatric: anemia, neutropenia, thrombocytopenia, and leukopenia ( 6.1 ) The most common (> 10%) neurological adverse reactions were: Adult: somnolence, dizziness, peripheral neurologic disorders, hypoesthesia, headache, and paresthesia ( 6.1 ) Pediatric: headache and peripheral neurologic disorders ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Alembic Pharmaceuticals, Inc. at 1-866-210-9797 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared with rates in the clinical trials of another drug and may not reflect the rates observed in practice. Relapsed or Refractory T-ALL and T-LBL Nelarabine injection was studied in 459 patients in Phase I and Phase II clinical trials.

Lactation : Advise not to breastfeed. ( 8.2 ) Renal Impairment : Closely monitor patients with moderate or severe renal impairment for toxicities. (8.6 ) Hepatic Impairment : Closely monitor patients with severe hepatic impairment for toxicities. ( 8.7 ) 8.1 Pregnancy Risk Summary Based on its mechanism of action and findings in animal studies, nelarabine injection can cause fetal harm when administered to a pregnant woman [see Clinical Pharmacology ( 12.1 )] . Limited available data with nelarabine injection use in pregnant women are insufficient to determine a drug-associated risk for major birth defects, miscarriage or adverse maternal or fetal outcomes. There are risks to the pregnant woman associated with untreated leukemia or lymphoma (see Clinical Considerations). In animal reproduction studies, intravenous administration of nelarabine to pregnant rabbits during the period of organogenesis resulted in teratogenicity at maternal doses below the recommended human adult dose of 1500 mg/m 2 /day (see Data) . Advise pregnant women of the potential risk to the fetus. The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S.

Metabolism

Absorption : Following intravenous administration of nelarabine to adult patients with refractory leukemia or lymphoma, plasma ara-G C max values generally occurred at the end of the nelarabine infusion and were generally higher than nelarabine C max values, suggesting rapid and extensive conversion of nelarabine to ara-G. Mean plasma nelarabine and ara-G C max values were 5.0 ± 3.0 mcg/mL and 31.4 ± 5.6 mcg/mL, respectively, after a 1,500 mg/m 2 nelarabine dose infused over 2 hours in adult patients.

There is no known antidote for overdoses of nelarabine injection. It is anticipated that overdosage would result in severe neurotoxicity (possibly including paralysis, coma), myelosuppression, and potentially death. In the event of overdose, supportive care consistent with good clinical practice should be provided. At a dose of 2,200 mg/m 2 given on Days 1, 3, and 5 every 21 days, 2 patients developed a significant Grade 3 ascending sensory neuropathy. MRI evaluations of the 2 patients demonstrated findings consistent with a demyelinating process in the cervical spine.

• Oct 28, 2005NDANDA021877Sandoz
• Nov 17, 2021ANDAANDA215037Zydus Pharms
• Dec 22, 2022ANDAANDA216934Dr Reddys
• Jan 10, 2023ANDAANDA216038Meitheal
• Mar 3, 2023ANDAANDA214809Shorla
• Apr 4, 2023ANDAANDA216346Amneal
• Jan 3, 2024ANDAANDA212605Gland
• Aug 1, 2024ANDAANDA218554Alembic

1. 1Febrile Neutropenia757.9%
2. 2Drug Ineffective636.6%
3. 3Neurotoxicity606.3%
4. 4Neuropathy Peripheral565.9%
5. 5Muscular Weakness555.8%
6. 6Sepsis555.8%
7. 7Peripheral Sensory Neuropathy545.7%
8. 8Myelopathy525.5%
9. 9Off Label Use505.3%
10. 10Neutropenia485.1%
11. 11Guillain-barre Syndrome424.4%
12. 12Hypoaesthesia394.1%
13. 13Gait Disturbance384.0%
14. 14Thrombocytopenia363.8%
15. 15Peripheral Motor Neuropathy353.7%

The 10 most recently updated of 33 ClinicalTrials.gov registrations naming Nelarabine as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Low-Intensity Chemotherapy and Venetoclax in Treating Patients With Relapsed or Refractory B- or T-Cell Acute Lymphoblastic Leukemia
  Terminated · Phase 1 · Phase 2 · Interventional · 53 enrolled · M.D. Anderson Cancer Center
  NCT03808610updated 2026-05-13
• Adding Dasatinib Or Venetoclax To Improve Responses In Children With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia (ALL) Or Lymphoma (T-LLY) Or Mixed Phenotype Acute Leukemia (MPAL)
  Recruiting · Phase 2 · Interventional · 100 enrolled · St. Jude Children's Research Hospital
  NCT06390319updated 2026-05-01
• CApivasertib, Venetoclax And Low-intensity chemotheRapY for Adults With ALL/LBL
  Recruiting · Phase 1 · Phase 2 · Interventional · 104 enrolled · University of Chicago
  NCT07294677updated 2026-03-04
• Treatment of Newly Diagnosed Acute Lymphoblastic Leukemia in Children and Adolescents
  Active not recruiting · Phase 3 · Interventional · 560 enrolled · Dana-Farber Cancer Institute
  NCT03020030updated 2026-01-22
• Combination Chemotherapy and Nelarabine in Treating Patients With T-cell Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma
  Recruiting · Phase 2 · Interventional · 160 enrolled · M.D. Anderson Cancer Center
  NCT00501826updated 2026-01-07
• Testing the Addition of Daratumumab to Chemotherapy for Treating Patients With Newly-Diagnosed T-Cell Lymphoblastic Leukemia (T-ALL) and T-Cell Lymphoblastic Lymphoma (T-LL)
  Not yet recruiting · Phase 2 · Phase 3 · Interventional · 1,708 enrolled · Children's Oncology Group
  NCT07072585updated 2025-12-31
• Optimization of Therapy in Adult Patients With Newly Diagnosed Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma by Individualised, Targeted and Intensified Treatment
  Completed · Phase 3 · Interventional · 1,023 enrolled · Goethe University
  NCT02881086updated 2025-12-16
• Total Therapy XVII for Newly Diagnosed Patients With Acute Lymphoblastic Leukemia and Lymphoma
  Active not recruiting · Phase 2 · Phase 3 · Interventional · 790 enrolled · St. Jude Children's Research Hospital
  NCT03117751updated 2025-12-05
• Combination Chemotherapy in Treating Young Patients With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia or T-cell Lymphoblastic Lymphoma
  Completed · Phase 3 · Interventional · 1,895 enrolled · National Cancer Institute (NCI)
  NCT00408005updated 2025-04-20
• Standard Chemotherapy with or Without Nelarabine or Rituximab in Treating Patients with Newly Diagnosed Acute Lymphoblastic Leukemia
  Completed · Interventional · University College, London
  NCT01085617updated 2025-03-30

Frequently asked questions

How does Nelarabine work?

Nelarabine is a prodrug of the deoxyguanosine analogue 9-β- D -arabinofuranosylguanine (ara-G), a nucleoside metabolic inhibitor. Nelarabine is demethylated by adenosine deaminase (ADA) to ara-G, mono-phosphorylated by deoxyguanosine kinase and deoxycytidine kinase, and subsequently converted to the active 5’-triphosphate, ara-GTP.

What is Nelarabine used for?

According to FDA labeling, Nelarabine carries indications including: Nelarabine injection is indicated for the treatment of T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL) in adult and pediatric patients age 1 year and older whose disease has not responded to or has relapsed following treatment with at least two chemotherapy regimens. Nelarabine injection is a nucleoside metabolic inhibitor indicated for the treatment of patients with T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma in adult and pediatric patients age 1 year and older whose disease has not responded to or has relapsed following treatment with at least two chemotherapy regimens.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Nelarabine?

Nelarabine is classified as Purine analogues, Nucleoside Metabolic Inhibitor, Nucleic Acid Synthesis Inhibitors, Nucleoside Synthesis Inhibitors, Decreased DNA Integrity, Decreased DNA Replication, Increased Cellular Death.

What are the brand names for Nelarabine?

Nelarabine is marketed under brand names including Arranon.

What are the contraindications for Nelarabine?

Nelarabine labeling lists contraindications including: None.. Always consult the full prescribing information and a clinician.