Treosulfan is a DNA alkylating agent used in the context of myeloablative conditioning prior to allogeneic hemoatopoietic stem cell transplantation. It was approved by the EMA in June 2019 and by the FDA in January 2025 for use in combination with fludarabine.
- Mechanism
- Curator reviewed · 1 reference
Treosulfan - specifically its active epoxide metabolites - function as a DNA alkylating agent, which is likely responsible for its cytotoxic activity. DNA alkylation involves the substitution of alkyl groups for hydrogen atoms on DNA, allowing for cross-linking within the DNA molecule resulting in cytotoxic, mutagenic, and carcinogenic effects.
- Primary indication
- Treosulfan is indicated in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adult and pediatric patients 1 year of age and older with acute myeloid leukemia or myelodysplastic...Curator reviewed · 4 structured indications
- Formula / weight
- C6H14O8S2 · 278.29 g/mol (avg)
- First approval
- Canada, 2021 · United States, 2025 · European Union, 2020
- Also known as
- Dihydroxybusulfan · L-threitol-1,4-dimethanesulfonate · Ovastat
- Code names
- NSC-39069
- Brand names
Resolves to
YCPOZVAOBBQLRI-WDSKDSINSA-NSMILESCS(=O)(=O)OC[C@H](O)[C@@H](O)COS(C)(=O)=OWhat you can answer from here — as of September 13, 2026
Which other approved drugs treat the same conditions as Treosulfan, and which companies have late-stage candidates in those indications?
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