Overview
Busulfan is a bifunctional alkylating agent, having a selective immunosuppressive effect on bone marrow. It is not a structural analog of the nitrogen mustards. It has been used in the palliative treatment of chronic myeloid leukemia (myeloid leukemia, chronic), but although symptomatic relief is provided, no permanent remission is brought about. According to the Fourth Annual Report on Carcinogens (NTP 85-002, 1985), busulfan is listed as a known carcinogen.
Indication
For use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous (myeloid, myelocytic, granulocytic) leukemia (FDA has designated busulfan as an orphan drug for this use). It is also used as a component of pretransplant conditioning regimens in patients undergoing bone marrow transplantation for acute myeloid leukemia and nonmalignant diseases.
Associated Conditions
- Chronic Myelogenous Leukemia (CML)
- Essential Thrombocythemia (ET)
- Polycythemia Vera (PV)
Research Report
Busulfan Report
Name: Busulfan Name (English): Busulfan DrugBank ID: DB01008 Type: Small Molecule CAS Number: 55-98-1
Description: Busulfan is an alkylating agent used primarily in the treatment of chronic myelogenous leukemia and as part of conditioning regimens before hematopoietic stem cell transplantation for various hematologic malignancies and non-malignant diseases. It works by alkylating DNA, which disrupts DNA replication and RNA transcription, ultimately leading to cell death.
Pharmacodynamics: Busulfan is a bifunctional alkylating agent that forms DNA crosslinks, mainly guanine-adenine intrastrand crosslinks, interfering with DNA replication and RNA transcription. This non-specific action affects both cancerous and normal cells, particularly hematopoietic progenitor cells.
Pharmacokinetics:
- Absorption: Well absorbed from the gastrointestinal tract, with an absolute bioavailability of approximately 80%. Peak plasma concentrations are reached within approximately 0.9 hours.
- Distribution: Highly lipophilic and crosses the blood-brain barrier.
- Metabolism: Extensively metabolized in the liver, primarily by conjugation with glutathione via glutathione S-transferases (GSTs), with GSTA1 being the primary isoform.
- Elimination: Metabolites are excreted in the urine.
Indications:
- Palliative treatment of chronic myelogenous leukemia.
- Conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia.
- Component of pretransplant conditioning regimens in patients undergoing bone marrow transplantation for acute myeloid leukemia and nonmalignant diseases.
Contraindications:
- Hypersensitivity to busulfan.
- Severe bone marrow depression.
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
|---|---|---|---|---|---|
2025/07/04 | Not Applicable | Not yet recruiting | |||
2025/05/30 | Phase 1 | Not yet recruiting | City of Hope Medical Center | ||
2025/05/07 | Phase 1 | ENROLLING_BY_INVITATION | |||
2025/05/02 | Phase 2 | Not yet recruiting | |||
2025/03/12 | Phase 2 | Recruiting | |||
2025/02/28 | Phase 1 | ENROLLING_BY_INVITATION | |||
2025/02/05 | Phase 1 | Recruiting | He Huang | ||
2025/02/05 | Phase 1 | Recruiting | He Huang | ||
2025/01/31 | Phase 2 | Recruiting | |||
2024/09/25 | Phase 3 | Not yet recruiting |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
|---|---|---|---|---|---|
| Accord Healthcare, Inc | 16729-351 | INTRAVENOUS | 6 mg in 1 mL | 1/5/2024 | |
| Meitheal Pharmaceuticals Inc. | 71288-158 | INTRAVENOUS | 6 mg in 1 mL | 4/21/2021 | |
| Sagent Pharmaceuticals | 25021-241 | INTRAVENOUS | 6 mg in 1 mL | 9/10/2020 | |
| Otsuka America Pharmaceutical, Inc. | 59148-070 | INTRAVENOUS | 6 mg in 1 mL | 3/25/2020 | |
| Mylan Institutional LLC | 67457-893 | INTRAVENOUS | 6 mg in 1 mL | 5/10/2017 | |
| Waylis Therapeutics LLC | 80725-620 | ORAL | 2 mg in 1 1 | 5/15/2023 | |
| Hospira, Inc. | 0409-1112 | INTRAVENOUS | 6 mg in 1 mL | 8/10/2020 | |
| Nexus Pharmaceuticals LLC | 14789-013 | INTRAVENOUS | 60 mg in 10 mL | 9/7/2023 | |
| Woodward Pharma Services LLC | 69784-620 | ORAL | 2 mg in 1 1 | 4/15/2022 | |
| Pharmascience Inc. | 51817-170 | INTRAVENOUS | 6 mg in 1 mL | 5/18/2022 |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
|---|---|---|---|
Authorised | 9/22/2014 |
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| BUSULFEX® INJECTION 60 mg/vial | SIN13260P | INJECTION, SOLUTION, CONCENTRATE | 60 mg/vial | 4/30/2007 | |
| MYRAN TABLETS 2 mg | SIN11001P | TABLET | 2 mg | 6/29/1999 |
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
|---|---|---|---|---|---|
| Busulfan Injection | 国药准字HJ20170309 | 化学药品 | 注射剂 | 3/3/2023 | |
| Busulfan Injection | 国药准字H20153304 | 化学药品 | 注射剂 | 2/3/2021 | |
| Busulfan Injection | 国药准字H20213017 | 化学药品 | 注射剂 | 1/12/2021 | |
| Busulfan Injection | 国药准字H20243505 | 化学药品 | 注射剂 | 4/17/2024 | |
| Busulfan Injection | 国药准字H20183147 | 化学药品 | 注射剂 | 3/23/2023 | |
| Busulfan Injection | 国药准字HJ20170308 | 化学药品 | 注射剂 | 3/3/2023 | |
| Busulfan Injection | 国药准字H20249359 | 化学药品 | 注射剂 | 11/15/2024 | |
| Busulfan Tablets | 国药准字H33020770 | 化学药品 | 片剂 | 1/7/2020 | |
| Busulfan Tablets | 国药准字H11020856 | 化学药品 | 片剂 | 11/27/2020 | |
| Busulfan Tablets | 国药准字H11020855 | 化学药品 | 片剂 | 11/27/2020 |
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
|---|---|---|---|---|---|
| No PPB approvals found for this drug. | |||||
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
|---|---|---|---|---|---|
| GenRx Busulfan busulfan 60mg/10mL injection vial | 210229 | Medicine | A | 10/9/2014 | |
| BUSULFAN ACCORD busulfan 60 mg/10 mL concentrated injection vial | 300848 | Medicine | A | 8/2/2019 | |
| APO-Busulfan busulfan 60mg/10mL injection vial | 210230 | Medicine | A | 10/9/2014 | |
| BUSULFEX busulfan 60mg/10mL injection vial | 150612 | Medicine | A | 7/21/2008 | |
| MYLERAN busulfan 2mg tablet bottle | 68028 | Medicine | A | 3/2/1999 | |
| Busulfan ARX busulfan 60mg/10mL injection vial | 210228 | Medicine | A | 10/9/2014 | |
| BUSULFAN INTAS busulfan 60 mg/10 mL concentrated injection vial | 300849 | Medicine | A | 8/2/2019 |
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