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BUSULFAN
BUSULFAN is a Alkylating Drug [EPC] drug. It is currently FDA-approved (first approved 1954).
Busulfan alkylates DNA, causing damage and cytotoxicity.
At a glance
| Generic name | BUSULFAN |
|---|---|
| Drug class | Alkylating Drug [EPC] |
| Modality | Small molecule |
| Phase | FDA-approved |
| First approval | 1954 |
Mechanism of action
Busulfan is a bifunctional alkylating agent that releases reactive carbonium ions when it hydrolyzes. These ions alkylate DNA, leading to DNA damage, which is believed to cause the drug's cytotoxic effects.
Approved indications
Boxed warnings
- WARNING: MYELOSUPPRESSION Busulfan injection causes severe and prolonged myelosuppression at the recommended dosage. Hematopoietic progenitor cell transplantation is required to prevent potentially fatal complications of the prolonged myelosuppression [see Warnings and Precautions (5.1) ]. WARNING: MYELOSUPPRESSION See full prescribing information for complete boxed warning. Causes severe and prolonged myelosuppression. ( 5.1 ) Hematopoietic progenitor cell transplantation is required to prevent potentially fatal complications of the prolonged myelosuppression. ( 5.1 )
Common side effects
- Fever
- Headache
- Nausea
- Stomatitis
- Vomiting
- Anorexia
- Diarrhea
- Abdominal Pain
- Insomnia
- Anxiety
- Hypomagnesemia
- Hyperglycemia
Key clinical trials
- Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies (PHASE2)
- Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease (PHASE1, PHASE2)
- Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID) (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)
- Base Editing Hematopoietic Stem Cell and T Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study (PHASE1, PHASE2)
- Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists (PHASE1, PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- BUSULFAN CI brief — competitive landscape report
- BUSULFAN updates RSS · CI watch RSS
Frequently asked questions about BUSULFAN
What is BUSULFAN?
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What are the side effects of BUSULFAN?
Related
- Drug class: All Alkylating Drug [EPC] drugs
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing