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intravenous busulfan
Intravenous busulfan is an alkylating agent that interferes with DNA replication, leading to cell death in rapidly dividing cells.
Intravenous busulfan is an alkylating agent that interferes with DNA replication, leading to cell death in rapidly dividing cells. Used for Acute myeloid leukemia (AML), Chronic myeloid leukemia (CML).
At a glance
| Generic name | intravenous busulfan |
|---|---|
| Sponsor | Alberta Health services |
| Drug class | Alkylating agent |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
Busulfan works by adding an alkyl group to the DNA of cancer cells, which damages the cells' ability to replicate and ultimately leads to cell death. This mechanism is particularly effective against rapidly dividing cells, such as those found in leukemia. As a result, busulfan is often used in chemotherapy regimens to treat various types of cancer.
Approved indications
- Acute myeloid leukemia (AML), Chronic myeloid leukemia (CML)
Common side effects
- Nausea
- Vomiting
- Anemia
- Thrombocytopenia
- Neutropenia
Key clinical trials
- Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype (PHASE3)
- Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease (PHASE1, PHASE2)
- Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease (PHASE3)
- Allogeneic Hematopoietic Cell Transplantation for Disorders of T-cell Proliferation and/or Dysregulation (PHASE2)
- A Platform Protocol to Investigate Post-Transplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxis in Patients With Hematologic Malignancies Undergoing Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation (PHASE2)
- A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD) (PHASE2)
- Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) (PHASE3)
- Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) (PHASE3)
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 |
|---|---|
| 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:
- intravenous busulfan CI brief — competitive landscape report
- intravenous busulfan updates RSS · CI watch RSS
- Alberta Health services portfolio CI