medac Gesellschaft für klinische Spezialpräparate mbH has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion for the approval of TRECONDI® (treosulfan) in combination with fludarabine as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation in adult patients with malignant and non-malignant diseases, and in paediatric patients older than one month with malignant diseases.
The CHMP opinion is essentially based on the results of the pivotal MC-FludT.14/L-Part II phase III study and the MC-FludT.17/M phase II study. The CHMP opinion will now be reviewed by the European Commission. Its decision will then take effect for all 28 EU Member States plus Liechtenstein, Iceland, and Norway.
Allogeneic haematopoietic stem cell transplantation (allo-HSCT) is the only potentially curative treatment option for many malignant and non-malignant diseases. It is imperative that transplantation be preceded by preparatory conditioning therapies. The standard in this respect is conditioning with high-dose, toxic myeloablative regimens which are not suitable for numerous at-risk groups. Research has therefore been conducted into so-called reduced-intensity conditioning therapies.
The treosulfan-based treatment is characterised by a high level of intensity and antileukaemic effect comparable to that of the myeloablative regimens with considerably reduced toxicity at the same time.
The positive CHMP opinion takes into account the current study data for TRECONDI® and confirms the efficacy and safety of the treosulfan-based conditioning regimen with its low toxicity profile. This will benefit at-risk groups in particular, who are excluded from the currently established myeloablative regimens.