Treosulfan Medicine 07 June 2022 Extended indication Extension of indication to include additional non-malignant transplant indications (non-malignant diseases in the paediatric population) Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Treosulfan Domain Hematology Reason of inclusion Indication extension IND Main indication Stem cell transplants Extended indication Extension of indication to include additional non-malignant transplant indications (non-malignant diseases in the paediatric population) Current proprietary name Already available biosimilars / generics Proprietary name Trecondi Manufacturer Medac Portfolio holder Mechanism of action Alkylating agent Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date April 2022 Expected Registration February 2023 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 1 times a day Dosage per administration 10 g/m2 References NCT02349906 Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Expected cost per patient per year Cost References farmacotherapeutisch kompas Additional remarks - Trecondi, 1.000mg poeder voor oplossing voor infusie: €135,15 - Trecondi, 5.000mg poeder voor oplossing voor infusie: € 670,33 Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks