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IV Busulfan
IV Busulfan is a Alkylating agent Small molecule drug developed by Assistance Publique - Hôpitaux de Paris. It is currently in Phase 2 development for Chronic myeloid leukemia. Also known as: Busulfan, Busulfex®.
Alkylating agent that interferes with DNA replication
Alkylating agent that interferes with DNA replication Used for Chronic myeloid leukemia.
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Baseline phase 2 → approval rate
+15.3pp
Industry-wide phase 2 drugs reach approval ~15.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Oncology Phase 2 attrition
-2.0pp
Oncology drugs have higher Phase 2-to-Phase 3 attrition than average — many fail to show OS benefit in larger studies.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2031–2034 | — |
| EMA | EU | 2032–2035 | +0.7 yr |
| MHRA | GB | 2032–2035 | +0.7 yr |
| Health Canada | CA | 2032–2036 | +0.9 yr |
| TGA | AU | 2032–2036 | +1.2 yr |
| PMDA | JP | 2032–2036 | +1.5 yr |
| NMPA | CN | 2033–2037 | +2.3 yr |
| MFDS | KR | 2032–2036 | +1.4 yr |
| CDSCO | IN | 2032–2037 | +1.8 yr |
| ANVISA | BR | 2033–2037 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | IV Busulfan |
|---|---|
| Also known as | Busulfan, Busulfex® |
| Sponsor | Assistance Publique - Hôpitaux de Paris |
| Drug class | Alkylating agent |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 2 |
Mechanism of action
Busulfan is a nitrogen mustard alkylating agent that forms cross-links in DNA, thereby inhibiting DNA replication and transcription. This leads to cell death, particularly in rapidly dividing cells such as cancer cells.
Approved indications
- Chronic myeloid leukemia
Common side effects
- Bone marrow suppression
- Nausea and vomiting
- Diarrhea
Key clinical trials
- Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies (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)
- Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype (PHASE3)
- US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies (PHASE1, PHASE2)
- Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide (EARLY_PHASE1)
- Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations (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 |
|---|---|
| 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:
- IV Busulfan CI brief — competitive landscape report
- IV Busulfan updates RSS · CI watch RSS
- Assistance Publique - Hôpitaux de Paris portfolio CI
Frequently asked questions about IV Busulfan
What is IV Busulfan?
How does IV Busulfan work?
What is IV Busulfan used for?
Who makes IV Busulfan?
Is IV Busulfan also known as anything else?
What drug class is IV Busulfan in?
What development phase is IV Busulfan in?
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Related
- Drug class: All Alkylating agent drugs
- Manufacturer: Assistance Publique - Hôpitaux de Paris — full pipeline
- Therapeutic area: All drugs in Oncology
- Indication: Drugs for Chronic myeloid leukemia
- Also known as: Busulfan, Busulfex®
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing