Last reviewed · How we verify
Treosulfan IV
Treosulfan IV is a Small molecule drug developed by IRCCS San Raffaele. It is currently in Phase 2 development.
Treosulfan IV is a small molecule that acts as a DNA cross-linking agent, classified as a cross-linking agent. It is being studied in clinical trials for various conditions, including Acute Lymphoblastic Leukemia, Myelofibrosis, and Acute Myeloid Leukemia, often in combination with allogenic transplantation.
-
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).
| 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 | Treosulfan IV |
|---|---|
| Sponsor | IRCCS San Raffaele |
| Modality | Small molecule |
| Phase | Phase 2 |
Approved indications
Common side effects
Key clinical trials
- MT2025-35 Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning Treosulfan and Fludarabine, With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases (PHASE2)
- Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Thiotepa in Treating Patients With Non-malignant Disorders (PHASE2)
- Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) (PHASE2)
- Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Total-Body Irradiation for the Treatment of Hematological Malignancies (PHASE2)
- Treosulfan-Based Versus Clofarabine-Based Conditioning Before Donor Hematopoietic Stem Cell Transplant for the Treatment of Myelodysplastic Syndromes or Acute Myeloid Leukemia (PHASE2)
- Effect of Moderate Renal Impairment and Race/Ethnicity on Treosulfan Pharmacokinetics (PHASE1)
- Safety and Efficacy of FT14 Conditioning for Allogeneic HSCT in Acute Myeloid Leukemia (PHASE2)
- Allogeneic Stem Cell Transplantation for Children and Adolescents With Acute Lymphoblastic Leukaemia (PHASE2, 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:
- Treosulfan IV CI brief — competitive landscape report
- Treosulfan IV updates RSS · CI watch RSS
- IRCCS San Raffaele portfolio CI
Frequently asked questions about Treosulfan IV
What is Treosulfan IV?
Who makes Treosulfan IV?
What development phase is Treosulfan IV in?
Related
- Manufacturer: IRCCS San Raffaele — full pipeline
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing