Burosumab Medicine 08 June 2021 Extended indication Extension of indication to include treatment of FGF23-related hypophosphataemia in tumour-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumours that cannot be curatively resected or localised in patients aged 1 year and over. Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Burosumab Domain Metabolism and Endocrinology Reason of inclusion Indication extension IND Main indication Other metabolism and Endocrinology Extended indication Extension of indication to include treatment of FGF23-related hypophosphataemia in tumour-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumours that cannot be curatively resected or localised in patients aged 1 year and over. Current proprietary name Already available biosimilars / generics Proprietary name Crysvita Manufacturer Kyowa Portfolio holder Mechanism of action Unknown Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Burosumab is een humane monoclonale anti-FGF23 antistof. Verwachte meerwaarde met name bij kinderen, ten opzichte van fosfaatsuppletie en calcitriol. Studies worden nu afgerond. Er lijkt sprake te zijn van een significante toename fysieke functies en vermindering van stijfheid. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP No Submission date January 2021 Expected Registration November 2021 Orphan drug Yes 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 Dosage per administration References 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 Additional remarks 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