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Treosulfan (Treo)
Treosulfan (Treo) is a Alkylating agent Small molecule drug developed by Federal Research Institute of Pediatric Hematology, Oncology and Immunology. It is currently FDA-approved for Conditioning agent for hematopoietic stem cell transplantation in pediatric patients with hematologic malignancies and non-malignant disorders.
Treosulfan is an alkylating agent that cross-links DNA, causing cell death and used primarily as a myeloablative conditioning agent before hematopoietic stem cell transplantation.
Treosulfan (Treo) is a small molecule used as a cross-linking agent that targets DNA. It is being studied in clinical trials for various conditions, including pediatric hematopoietic stem cell transplantation, malignant and non-malignant disorders, acute myeloid leukemia, and myelodysplastic syndrome.
At a glance
| Generic name | Treosulfan (Treo) |
|---|---|
| Sponsor | Federal Research Institute of Pediatric Hematology, Oncology and Immunology |
| Drug class | Alkylating agent |
| Target | DNA |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
Mechanism of action
Treosulfan is a prodrug that is metabolized to active diepoxide metabolites, which alkylate DNA by forming inter- and intra-strand cross-links. This leads to cell cycle arrest and apoptosis. It is used as a conditioning regimen in hematopoietic stem cell transplantation, particularly in pediatric patients, due to its reduced toxicity profile compared to traditional alkylating agents like busulfan.
Approved indications
- Conditioning agent for hematopoietic stem cell transplantation in pediatric patients with hematologic malignancies and non-malignant disorders
Common side effects
- Myelosuppression
- Mucositis
- Nausea and vomiting
- Hepatotoxicity
- Infection
Key clinical trials
- Study of Treosulfan-Based Conditioning for HSCT in Nijmegen Breakage Syndrome (PHASE2)
- Evaluation of Treosulfan Versus Melphalan Conditioning Followed by PTCy in Patients With AML and MDS Undergoing Allogeneic Transplantation (PHASE2)
- Treosulfan Therapeutic Drug Monitoring in Pediatric Hematopoietic Stem Cell Transplant Recipients
- Allogeneic Stem Cell Transplantation for Children and Adolescents With Acute Lymphoblastic Leukaemia (PHASE2, PHASE3)
- Treo/Flu/TBI With Donor Stem Cell Transplant for Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia (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:
- Treosulfan (Treo) CI brief — competitive landscape report
- Treosulfan (Treo) updates RSS · CI watch RSS
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology portfolio CI
Frequently asked questions about Treosulfan (Treo)
What is Treosulfan (Treo)?
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Related
- Drug class: All Alkylating agent drugs
- Target: All drugs targeting DNA
- Manufacturer: Federal Research Institute of Pediatric Hematology, Oncology and Immunology — full pipeline
- Therapeutic area: All drugs in Oncology
- Indication: Drugs for Conditioning agent for hematopoietic stem cell transplantation in pediatric patients with hematologic malignancies and non-malignant disorders
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing