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Busulphan plus Fludarabine
Busulphan plus Fludarabine is a Alkylating agent + Antimetabolite combination Small molecule drug developed by Gruppo Italiano Trapianto di Midollo Osseo. It is currently in Phase 3 development for Conditioning regimen for allogeneic hematopoietic stem cell transplantation in hematologic malignancies, Acute myeloid leukemia, Acute lymphoblastic leukemia. Also known as: I.V. BuFlu.
Busulphan and Fludarabine together act as a myeloablative conditioning regimen that destroys bone marrow cells to prepare patients for hematopoietic stem cell transplantation.
Busulphan and Fludarabine together act as a myeloablative conditioning regimen that destroys bone marrow cells to prepare patients for hematopoietic stem cell transplantation. Used for Conditioning regimen for allogeneic hematopoietic stem cell transplantation in hematologic malignancies, Acute myeloid leukemia, Acute lymphoblastic leukemia.
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Baseline phase 3 → approval rate
+58.3pp
Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Oncology Phase 3 boost
+3.0pp
Oncology Phase 3 trials have higher approval rates (~61%) than the cross-industry average due to clearer endpoints and FDA oncology pathway.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2028–2030 | — |
| EMA | EU | 2029–2031 | +0.7 yr |
| MHRA | GB | 2029–2031 | +0.7 yr |
| Health Canada | CA | 2029–2032 | +0.9 yr |
| TGA | AU | 2029–2032 | +1.2 yr |
| PMDA | JP | 2029–2032 | +1.5 yr |
| NMPA | CN | 2030–2033 | +2.3 yr |
| MFDS | KR | 2029–2032 | +1.4 yr |
| CDSCO | IN | 2029–2033 | +1.8 yr |
| ANVISA | BR | 2030–2033 | +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 | Busulphan plus Fludarabine |
|---|---|
| Also known as | I.V. BuFlu |
| Sponsor | Gruppo Italiano Trapianto di Midollo Osseo |
| Drug class | Alkylating agent + Antimetabolite combination |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
Busulphan is an alkylating agent that cross-links DNA and causes cell death, while Fludarabine is a purine analog that inhibits DNA synthesis and repair. When combined, they provide intensive cytotoxic conditioning to eliminate malignant and host bone marrow cells, allowing engraftment of donor stem cells in transplant recipients. This combination is used as a preparative regimen prior to allogeneic hematopoietic stem cell transplantation.
Approved indications
- Conditioning regimen for allogeneic hematopoietic stem cell transplantation in hematologic malignancies
- Acute myeloid leukemia
- Acute lymphoblastic leukemia
- Chronic myeloid leukemia
- Myelodysplastic syndrome
Common side effects
- Myelosuppression
- Mucositis
- Nausea and vomiting
- Hepatotoxicity
- Infection
- Graft-versus-host disease
- Hemorrhagic cystitis
Key clinical trials
- 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)
- US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies (PHASE1, PHASE2)
- Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations (PHASE2)
- Ruxolitinib-Decitabine Intensified Conditioning Regimen for AML: A Randomized Trial (PHASE4)
- HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation With Post-Transplantation Cyclophosphamide (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)
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:
- Busulphan plus Fludarabine CI brief — competitive landscape report
- Busulphan plus Fludarabine updates RSS · CI watch RSS
- Gruppo Italiano Trapianto di Midollo Osseo portfolio CI
Frequently asked questions about Busulphan plus Fludarabine
What is Busulphan plus Fludarabine?
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What is Busulphan plus Fludarabine used for?
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Is Busulphan plus Fludarabine also known as anything else?
What drug class is Busulphan plus Fludarabine in?
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Related
- Drug class: All Alkylating agent + Antimetabolite combination drugs
- Manufacturer: Gruppo Italiano Trapianto di Midollo Osseo — full pipeline
- Therapeutic area: All drugs in Oncology
- Indication: Drugs for Conditioning regimen for allogeneic hematopoietic stem cell transplantation in hematologic malignancies
- Indication: Drugs for Acute myeloid leukemia
- Indication: Drugs for Acute lymphoblastic leukemia
- Also known as: I.V. BuFlu
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing