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Treosulfan: Cytotoxic Drug Stability Monograph

11 February 2021Stability information is essential for the safe aseptic preparation of treosulfan
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New Medicines

Trecondiin combination with fludarabine as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in adult patients with malignant and non-malignant diseases, and in paediatric patients >1 month with malignant diseases.

Information

Trecondi
New formulation and new indication
Medac
Medac

Development and Regulatory status

Launched
Launched
Not approved
July 2020
Aug 21Medexus Pharmaceuticals and medac GmbH received a Complete Response Letter (CRL) from the US FDA. The response letter indicated that it couldn't be approved in its present form. It offered specific recommendations, including additional clinical and statistical data related to the primary and secondary endpoints of the pivotal PIII trial. The companies say they are reviewing the CRL to decide on a course of action. [12]
Aug 20Filed in US.[11]
Jul 20Launched in the UK. List price is £494.40 for 5 x 1g , and £2,434.25 for 5 x 5g vials [9,10]
Aug 19Launched in Germany [7].
Jun 19Approved in EU [6].
Jun 19The EMA Committee for Orphan Medicinal Products recommends that Trecondi, treosulfan (EU/3/04/186) for conditioning treatment prior to haematopoietic progenitor cell transplantation is removed from the Community Register of Orphan Medicinal Products [8].
Dec 18Recommended for EU approval by CHMP - the full indication is "Treosulfan in combination with fludarabine is indicated as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in adult patients with malignant and non-malignant diseases, and in paediatric patients older than one month with malignant diseases.” It is proposed that it be prescribed by physicians experienced in conditioning treatment followed by alloHSCT [5].
Oct 18Has orphan drug status in EU [3].
Feb 18Filed in EU [4].

Category

Alkylating agent
The latest available statistics show that in 2012 there were 1,361 first allograft transplants, including 718 for leukaemia, and 208 for MDS or mucopolysaccharide diseases (MPS), and 211 for lymphoma [1].
in combination with fludarabine as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in adult patients with malignant and non-malignant diseases, and in paediatric patients >1 month with malignant diseases.
Intravenous infusion

Further information

Yes

Trial or other data

Dec 17PIII study (NCT00822393) completes [2].
Nov 08PIII trial comparing the treosulfan-based conditioning therapy, with reduced-intensity busulfan-based conditioning therapy, prior to allogeneic haematopoietic stem cell transplantation, in patients with acute myeloid leukaemia or myelodysplastic syndromes starts (NCT00822393). 570 adults will be recruited in Germany, Finland, France, Hungary, Italy and Poland. Collection of primary outcome data (event-free survival) is due to complete Dec 17 [2].

Evidence based evaluations