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Drug

Treosulfan

Approved · EMA
Late-stage development

Also known as Dihydroxybusulfan, Grafapex, Treosulphan, L-threitol-1,4-dimethanesulfonate.

RxNorm38508ChEMBLCHEMBL455186UNIICO61ER3EPI
9
Active clinical trials
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

Hematopoietic Stem Cell Transplantation From Haploidentical Donors in Patients With Hematological Malignancies Using a Treosulfan-Based Preparative Regimen

Clinical trial2026-05-29Results expected Q2 2027 · ClinicalTrials.gov

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

Clinical trial2026-04-16Results expected Q1 2030 · ClinicalTrials.gov

Allogeneic Hematopoietic Cell Transplantation for Patients With Non-Malignant Disorders Using Treosulfan, Fludarabine, and Thiotepa

Clinical trial2026-03-12Results expected Q3 2028 · ClinicalTrials.gov

Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases

Clinical trial2026-02-18Completed · ClinicalTrials.gov

A Phase II Randomized Study to Assess Outcomes With Treosulfan-Based Versus Clofarabine-Based Conditioning in Patients With Myelodysplastic Syndromes With Excess Blasts (MDS-EB), or Acute Myeloid Leukemia (AML) Undergoing Allogeneic Hematopoietic Cell Transplantation (HCT)

Clinical trial2026-01-22Primary completion · ClinicalTrials.gov

A Clinical Study of the Efficacy and Safety of Conditioning With Low Doses of Treosulfan Before Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Nijmegen Breakage Syndrome

Clinical trial2026-01-05Results expected Q4 2028 · ClinicalTrials.gov

Randomized Evaluation of Treosulfan Versus Melphalan Conditioning Followed by PTCy in Patients With AML and MDS Undergoing Allogeneic Transplantation

Clinical trial2025-09-03Results expected Q4 2028 · ClinicalTrials.gov

An Open-label, Non-randomized, Prospective Trial to Evaluate the Effect of Renal Function Impairment and Race/Ethnicity on Treosulfan Pharmacokinetics in Patients With AML or MDS Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Clinical trial2025-06-13Completed · ClinicalTrials.gov

Allogeneic Stem Cell Transplantation for Children and Adolescents With Acute Lymphoblastic Leukaemia

Clinical trial2025-06-01Primary completion · ClinicalTrials.gov

A Randomized Phase II Trial Comparing Treosulfan and Melphalan With Melphalan Alone as Conditioning Regimen for Autologous Stem Cell Transplantation (ASCT) in Myeloma Patients (TreoMel Trial)

Clinical trial2024-07-08Results expected Q1 2028 · ClinicalTrials.gov

Clinical Open-label Phase 2 Study of Low Dose Treosulfan Based Conditioning Regimen Efficacy in Hematopoietic Stem Cell Transplantation With Post-transplant Cyclophosphamide for Children Nijmegen Breakage Syndrome

Clinical trial2024-01-10Results expected Q1 2027 · ClinicalTrials.gov

Approval: Trecondi (EMA)

Regulatory2019-06-20EMA

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Treosulfan
Aliases & brands
DihydroxybusulfanGrafapexTreosulphanL-threitol-1,4-dimethanesulfonate
RxNorm CUI
38508
ChEMBL ID
CHEMBL455186
UNII
CO61ER3EPI
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2019-06-20
Latest approval
2019-06-20
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2019-06-20Approval
Approval: Trecondi (EMA)
Indication: Treosulfan in combination with fludarabine is indicated as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in adult… Show full indication

Treosulfan in combination with fludarabine is indicated as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in adult patients and in paediatric patients older than one month with malignant and non-malignant diseases.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

34 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
34
registered trials across all phases
LATEST COMPLETION 2026
9
Active studies
8
Recruiting
4
Late-stage (III+)
17
Completed
8
Discontinued
PHASE DISTRIBUTIONn = 34
Phase 11Phase 1 / 22Phase 227Phase 2 / 31Phase 33

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

Allogeneic Stem Cell Transplantation for Children and Adolescents With Acute Lymphoblastic Leukaemia

RecruitingPH II/IIIKey trialSt. Anna Kinderkrebsforschungn=1,8002030

Phase 3, Open Label, Multi-centre, Randomised Controlled International Study in Ewing Sarcoma

CompletedPH IIIUniversity Hospital Muenstern=9072019

Clinical Phase III Trial to Compare Treosulfan-based Conditioning Therapy With Busulfan-based Reduced-intensity Conditioning (RIC) Prior to Allogeneic Haematopoietic Stem Cell Transplantation in Patients With AML or MDS Considered Ineligible to Standard Conditioning Regimens

CompletedPH IIImedac GmbHn=5702018

Präferenz-Studie Bei älteren Patientinnen Mit Ovarialkarzinomrezidiv: Treosulfan Oral vs. intravenös

CompletedPH IIINorth Eastern German Society of Gynaecological Oncologyn=1232012

Recent completions

Trials that read out recently, adding to the completed evidence base.

Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases

CompletedPH IIFred Hutchinson Cancer Centern=402026

An Open-label, Non-randomized, Prospective Trial to Evaluate the Effect of Renal Function Impairment and Race/Ethnicity on Treosulfan Pharmacokinetics in Patients With AML or MDS Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

CompletedPH Imedac GmbHn=202025

Clinical Phase II Trial to Compare Treosulfan-based Conditioning Therapy With Busulfan-based Conditioning Prior to Allogeneic Haematopoietic Stem Cell Transplantation (HSCT) in Paediatric Patients With Non-malignant Diseases

CompletedPH IImedac GmbHn=1062023

Evaluation of Safety and Efficacy of Treosulfan-cytarabine-fludarabine (FLAT) Combination Prior to Autologous Stem Cell Transplant (HSCT) in Elderly Patients With Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)

CompletedPH IICiceri Fabion=152021

Transplantation of Umbilical Cord Blood in Patients With Hematological Malignancies Using a Treosulfan Based Preparative Regimen

CompletedPH IIFred Hutchinson Cancer Centern=1302021

Allogeneic Hematopoietic Cell Transplantation for Patients With Nonmalignant Inherited Disorders Using a Treosulfan Based Preparative Regimen

CompletedPH IIFred Hutchinson Cancer Centern=982020
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.