Drug Profile


Alternative Names: CB 2562; CB 40067; CCRIS 2781; Dihydroxybusulphan; HSDB 6963; NSC 39069; Ovastat

Latest Information Update: 28 Jul 2017

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At a glance

  • Originator Medac
  • Developer Center for International Blood and Marrow Transplant Research; Medac
  • Class Antineoplastics; Butylene glycols; Mesylates; Small molecules
  • Mechanism of Action Alkylating agents; DNA cross linking agents; DNA synthesis inhibitors
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Transplant rejection
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Ovarian cancer
  • Phase III Acute myeloid leukaemia; Myelodysplastic syndromes
  • Phase II Acute lymphoblastic leukaemia; Haemoglobinopathies; Immunodeficiency disorders; Inborn error metabolic disorders
  • No development reported Renal cell carcinoma; Small cell lung cancer; Solid tumours
  • Discontinued Malignant melanoma

Most Recent Events

  • 28 Jul 2017 Treosulfan is still in phase II trials for Haemoglobinopathies, Immunodeficiency disorders, and Inborn error metabolic disorders (In adolescents, In children, In infants) in Poland, Italy, Czech Republic, Austria and Germany (IV) (NCT02349906)
  • 15 Mar 2016 Biomarkers information updated
  • 01 Aug 2015 Medac completes a phase II trial in Acute Myeloid leukaemia and Myelodysplastic syndrome (in infants, children and adoloscents) in USA (NCT01772953)
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