Treosulfan

Articles · Lactation Safety Information · New Medicines ·

326774002

Articles

Lactation Safety Information

No published evidence of safety
Serious adverse effects reported in adults

New Medicines

Trecondi · Malignant disease and non-malignant disease - in combination with fludarabine prior to haematopoietic stem cell transplant

Information

Trecondi
New formulation and new indication
Medac
Medac

Development and Regulatory status

Approved (Licensed)
Launched
None
Aug 19 · Launched in Germany [7].
Jun 19 · Approved in EU [6].
Jun 19 · The 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 18 · Recommended 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 18 · Has orphan drug status in EU [3].
Feb 18 · Filed 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].
Malignant disease and non-malignant disease - in combination with fludarabine prior to haematopoietic stem cell transplant
Intravenous infusion

Further information

Yes
To be confirmed

Trial or other data

Dec 17 · PIII study (NCT00822393) completes [2].
Nov 08 · PIII 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