Burosumab Medicine 09 January 2018 Extended indication X-linked hypophosphatemia Therapeutic value Possible added value Total cost Registration phase Unknown Product Active substance Burosumab Domain Metabolism and Endocrinology Reason of inclusion Main indication Metabolic diseases Extended indication X-linked hypophosphatemia 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 Extramural (GVS) 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 vanstijfheid. Risico's: kosten. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date January 2017 Expected Registration February 2018 Orphan drug Yes Registration phase Unknown Reimbursement Medicine sluice Additional remarks Positieve opinie CHMP december 2017. Therapeutic value Current treatment options Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Met name bij kinderen, ten opzichte van fosfaatsuppletie en calcitriol. Duration of treatment Frequency of administration Dosage per administration References Additional remarks Expected patient volume per year Patient volume < 850 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Orphanet: prevalence of approximately 1/ 20,000. Dit betekent 850 per 17miljoen inwoners. Expected cost per patient per year Cost References Additional remarks Waarschijnlijk hoge kosten 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