Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
treosulfan
- Development stage
- Approved
- Lead developer
- medac
- Modality
- DNA Intercalators/Alkylators → Nucleic Acid-Directed Small Molecules → Small Molecules
- Administration
- Intravenous
01
Overview
Treosulfan is a small molecule, DNA alkylating agent used primarily as part of a myeloablative conditioning regimen prior to allogeneic hematopoietic stem cell transplantation (alloHSCT) in adults and pediatric patients aged 1 year and older with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). It acts as a prodrug that is converted under physiological conditions into active epoxide metabolites, which alkylate DNA, leading to cross-linking within the DNA molecule. This results in cytotoxicity by preventing cell division and inducing cell death. Treosulfan exhibits immunosuppressive effects by depleting hematopoietic stem cells and reducing cellularity of lymphatic organs. It was approved by the EMA in June 2019 and by the FDA in January 2025 for use in combination with fludarabine[1][3][4][7].
- Brand names
- GrafapexTrecondiOvastat
- Other names
- dihydroxybusulfanL-threitol-1,4-dimethanesulfonate
02
Targets
DNA
Beyond the preview
Go deeper on treosulfan.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on treosulfan.