OnCo
drugsProductNot mapped hereapproved

Treosulfan

A conditioning chemotherapy given before donor stem-cell transplant in AML and MDS; it was long used in Europe and reached the US in 2025.

MC-FludT.14/L (Lancet Haematol 2020): treosulfan-fludarabine improved event-free and overall survival versus reduced-intensity busulfan-fludarabine in older/comorbid AML/MDS patients. Also used in paediatric transplant and in ovarian cancer historically (EU).

Loading structure…
|
Treosulfan
PubChem
Modality
Alkylating conditioning agent (busulfan analogue)
Mechanism
Prodrug converting non-enzymatically to epoxides that alkylate DNA; myeloablative with lower hepatic and neurotoxicity than busulfan.
Brand / code
Grafapex
Medicare
Part B (clinician-administered)

Given by infusion or injection in a clinic or hospital outpatient department, so it is a Part B drug: Medicare pays 80% after the Part B deductible and the patient owes 20% coinsurance, uncapped in Original Medicare unless a Medigap policy applies. Transplant conditioning given inpatient; paid within the Part A transplant stay.

Commercial insurance
covered with prior authorisation

Bundled into the transplant authorisation; centres seek approval as part of the allogeneic transplant episode.

Sources: Medicare.gov: Chemotherapy · Medicare.gov: Prescription drugs (outpatient, Part B) · Medicare.gov: Inpatient hospital care (Part A). Not medical or financial advice; verify with your plan.

Not yet researchedSearch NICE
Notes
EU-licensed 2019 as conditioning before allogeneic transplant; used in UK paediatric protocols. NICE position not yet researched.

Sources: NICE search: treosulfan. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.

Regulatory

2top

Approvals

RegionYearIndication
EU2019Conditioning before allogeneic HSCT (with fludarabine) in adults and children
US2025Preparative regimen for allogeneic HSCT in AML/MDS with fludarabine

Trials

top
ClinicalTrials.gov · phase 2/3
refreshed 2026-09-09
51 studies11 recruiting43 Phase 210 Phase 3
Search “Treosulfan” on ClinicalTrials.gov →
Counts are from a name search and may include unrelated studies; up to 100 studies are summarised.

Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov
Treosulfan
intervention: Treosulfan
Open on ClinicalTrials.gov →

Country and place are remembered in this browser only. A postcode is sent to OpenStreetMap's Nominatim service to find coordinates when you press the button; nothing else leaves your device.

Latest papers

top
Latest papers · live from Europe PMC
Open in Europe PMC

Query for this drug: (TITLE:"Treosulfan" OR ABSTRACT:"Treosulfan" OR TITLE:"Grafapex" OR ABSTRACT:"Grafapex") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Treosulfan, not a curated reading list.

Connected

5top