Burosumab Medicine 10 June 2020 Extended indication Extension of indication to include treatment of adults with X-linked hypophosphataemia (XLH), and modification of the currently approved indication in children and adolescents, by removing the qualification ‘with growing skeletons’, in order to include treatment in all children with radiographic evidence of bone disease. Therapeutic value No judgement Total cost € 3,100,000.00 Registration phase Registration application pending Product Active substance Burosumab Domain Metabolism and Endocrinology Reason of inclusion Indication extension IND Main indication Metabolic diseases Extended indication Extension of indication to include treatment of adults with X-linked hypophosphataemia (XLH), and modification of the currently approved indication in children and adolescents, by removing the qualification ‘with growing skeletons’, in order to include treatment in all children with radiographic evidence of bone disease. Current proprietary name Already available biosimilars / generics Proprietary name Crysvita Manufacturer Kyowa Portfolio holder Mechanism of action Other, see general comments Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Burosumab is een humane monoclonale anti-FGF23 antistof. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date November 2019 Expected Registration October 2020 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Fosfaatsuppletie en vitamine D. Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Er is een meerwaarde aangetoond bij kinderen, ten opzichte van fosfaatsuppletie en vitamine D. Studies bij volwassenen tonen een significante toename van de fosfaat spiegel, herstel van fracturen en pseudofracturen en een wisselend effect op pijn en loopvermogen. Duration of treatment Frequency of administration 1 times a month Dosage per administration 1.0 mg/kg References NCT02537431 Additional remarks Expected patient volume per year Patient volume < 10 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Orphanet Additional remarks Dit gaat alleen om XLH: een speciale vorm van erfelijke rachitis (X-gebonden). Orphanet geeft hiervoor een prevalentie van 1-9 op 1.000.000. Dit zou betekenen dat er maximaal 150 patiënten in Nederland zijn. De verwachting is dat maximaal 10 patienten in aanmerking zullen komen voor burosumab. Expected cost per patient per year Cost € 310,000.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References Prestatie- en tariefbeschikking Add-ongeneesmiddelen, juli 2018 Additional remarks Een flacon 10mg/ml flacon 1ml kost €3.591,28, en een flacon 30mg/ml flacon 1ml kost €10.772,78. Een patiënt van 70 kg heeft 70 mg Crysvita per toediening nodig. Dat is in totaal €25.136,84. Voor een behandeling van een jaar (12 toedieningen) is dit in totaal €310.642,08. Potential total cost per year Total cost € 3,100,000.00 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