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Treosulfan

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Treosulfan
Clinical data
Trade namesTrecondi, others
Other names1,2,3,4-Butanetetrol, 1,4-dimethanesulfonate, Threitol 1,4-dimethanesulfonate, Threitol 1,4-bismethanesulfonate; L-Threitol 1,4-bis(methanesulfonate); Threosulphan; Treosulphan; Tresulfan
AHFS/Drugs.comInternational Drug Names
License data
Pregnancy
category
Routes of
administration		By mouth, intravenous
ATC code
Legal status
Legal status
Identifiers
  • (2S,3S)-2,3-Dihydroxybutane-1,4-diyl dimethanesulfonate
CAS Number
PubChem CID
DrugBank
ChemSpider
UNII
KEGG
ChEBI
CompTox Dashboard (EPA)
ECHA InfoCard100.005.529 Edit this at Wikidata
Chemical and physical data
FormulaC6H14O8S2
Molar mass278.29 g·mol−1
3D model (JSmol)
Melting point101.5 to 105 °C (214.7 to 221.0 °F)
  • CS(=O)(=O)OC[C@@H]([C@H](COS(=O)(=O)C)O)O
  • InChI=1S/C6H14O8S2/c1-15(9,10)13-3-5(7)6(8)4-14-16(2,11)12/h5-8H,3-4H2,1-2H3/t5-,6-/m0/s1
  • Key:YCPOZVAOBBQLRI-WDSKDSINSA-N
 ☒NcheckY (what is this?)  (verify)

Treosulfan, sold under the brand name Trecondi among others, is an alkylating medication given to people before they have a bone marrow transplant from a donor known as allogeneic hematopoietic stem cell transplantation. It is used as a 'conditioning' treatment to clear the bone marrow and make room for the transplanted bone marrow cells, which can then produce healthy blood cells.[9][10] It is used together with another medicine called fludarabine in adults and children from one month of age with blood cancers as well as in adults with other severe disorders requiring a bone marrow transplant.[9] It belongs to the family of drugs called alkylating agents.[9] In the body, treosulfan is converted into other compounds called epoxides which kill cells, especially cells that develop rapidly such as bone marrow cells, by attaching to their DNA while they are dividing.[9]

The most common side effects include infections, nausea (feeling sick), stomatitis (inflammation of the lining of the mouth), vomiting, diarrhea, and abdominal pain (belly ache).[9] Tiredness, febrile neutropenia (low white blood cell counts with fever) and high blood levels of bilirubin (a breakdown product of red blood cells) are also seen in more than 1 in 10 adults, and rash also affects more than 1 in 10 children.[9] The most common adverse reactions include musculoskeletal pain, stomatitis, pyrexia, nausea, edema, infection, and vomiting.[7] Selected grade 3 or 4 nonhematological laboratory abnormalities include increased GGT, increased bilirubin, increased ALT, increased AST, and increased creatinine.[7]

Treosulfan was authorized for medical use in the European Union in June 2019,[9] and approved for medical use in the United States in January 2025.[7][11]

Medical Uses

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Treosulfan in combination with fludarabine is indicated as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation in adults with malignant and non malignant diseases, and in children older than one month with malignant diseases.[7][9]

History

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Two main studies showed that treosulfan is at least as effective as busulfan, another medicine used to prepare people for haematopoietic stem cell transplantation.[9]

In one of the studies, involving 570 adults with acute myeloid leukaemia (a blood cancer) or myelodysplastic syndromes (conditions in which large numbers of abnormal blood cells are produced), 64% of patients given treosulfan (with fludarabine) had a successful transplant and were alive and disease-free after 2 years, compared with 51% of patients given busulfan (with fludarabine).[9]

In an additional study in 70 children with blood cancers, 99% of children given treosulfan (with fludarabine) were alive three months after their transplant.[9]

Efficacy was evaluated in MC-FludT.14/L Trial II (NCT00822393), a randomized active-controlled trial comparing treosulfan to busulfan with fludarabine as a preparative regimen for allogeneic transplantation. Eligible patients included adults 18 to 70 years old with AML or MDS, Karnofsky performance status ≥ 60%, and age ≥ 50 years or hematopoietic cell transplantation comorbidity index [HCTCI] score > 2. There were 570 patients randomized to treosulfan (n=280) or busulfan (n=290).

Society and culture

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Treosulfan was authorized for medical use in the European Union in June 2019,[9] and approved for medical use in the United States in January 2025.[11][12][13]

The US Food and Drug Administration granted orphan drug designation to treosulfan in 1994, for the treatment of ovarian cancer;[14] and in 2015, for conditioning treatment prior to hematopoietic stem cell transplantation in malignant and non-malignant diseases in adults and pediatric patients.[15]

In February 2004, orphan designation (EU/3/04/186) was granted by the European Commission to medac Gesellschaft fuer klinische Spezialpräparate mbH, Germany, for treosulfan for the conditioning treatment prior to haematopoietic progenitor cell transplantation.[16]

Names

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Treosulfan is the international nonproprietary name.[17]

Treosulfan is sold under the brand names Trecondi[9] and Grafapex.[7]

References

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  1. ^ a b "Trecondi APMDS". Therapeutic Goods Administration (TGA). 11 October 2022. Retrieved 25 January 2025.
  2. ^ "Updates to the Prescribing Medicines in Pregnancy database". Therapeutic Goods Administration (TGA). 21 December 2022. Archived from the original on 3 April 2022. Retrieved 2 January 2023.
  3. ^ "Trecondi (Link Medical Products Pty Ltd T/A Link Pharmaceuticals)". Therapeutic Goods Administration (TGA). 14 January 2025. Retrieved 25 January 2025.
  4. ^ "AusPAR: Trecondi". Therapeutic Goods Administration (TGA). 4 July 2023. Retrieved 25 January 2025.
  5. ^ "Health product highlights 2021: Annexes of products approved in 2021". Health Canada. 3 August 2022. Retrieved 25 March 2024.
  6. ^ "Treosulfan 5g Powder for Solution for Infusion - Summary of Product Characteristics (SmPC)". (emc). Archived from the original on 20 May 2022. Retrieved 21 April 2020.
  7. ^ a b c d e f https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/214759s000lbl.pdf
  8. ^ "Trecondi Product Information" (PDF). European Medicines Agency (EMA). 21 April 2020.
  9. ^ a b c d e f g h i j k l m "Trecondi EPAR". European Medicines Agency (EMA). 11 December 2018. Archived from the original on 16 March 2023. Retrieved 21 April 2020. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  10. ^ Romański M, Wachowiak J, Główka FK (October 2018). "Treosulfan Pharmacokinetics and its Variability in Pediatric and Adult Patients Undergoing Conditioning Prior to Hematopoietic Stem Cell Transplantation: Current State of the Art, In-Depth Analysis, and Perspectives". Clinical Pharmacokinetics. 57 (10): 1255–1265. doi:10.1007/s40262-018-0647-4. PMC 6132445. PMID 29557088.
  11. ^ a b "FDA approves treosulfan with fludarabine as a preparative regimen for alloHSCT in adult and pediatric patients with AML or MDS". U.S. Food and Drug Administration (FDA). 6 February 2025. Retrieved 8 March 2025. Public Domain This article incorporates text from this source, which is in the public domain.
  12. ^ "Novel Drug Approvals for 2025". U.S. Food and Drug Administration (FDA). 21 February 2025. Retrieved 9 March 2025.
  13. ^ "Medexus Announces FDA Approval of Grafapex (treosulfan) for Injection and Provides Business Update" (Press release). Medexus Pharmaceuticals. 22 January 2025. Retrieved 25 January 2025 – via Newsfile.
  14. ^ "Treosulfan Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 16 May 1994. Retrieved 9 March 2025.
  15. ^ "Treosulfan Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 8 April 2015. Retrieved 9 March 2025.
  16. ^ "EU/3/04/186". European Medicines Agency (EMA). 17 September 2018. Archived from the original on 16 October 2019. Retrieved 21 April 2020. Public Domain This article incorporates text from this source, which is in the public domain.
  17. ^ World Health Organization (1972). "International nonproprietary names for pharmaceutical substances (INN). recommended INN: list 12". WHO Chronicle. 26 (10).
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  1. ^ "Treosulfan (Code C1257)". NCI Thesaurus. 25 November 2024. Retrieved 9 March 2025.
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