Roluperidon

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
Registration phase	Registration application pending

Product

Active substance	Burosumab
Domain	Metabolism and Endocrinology
Reason of inclusion	Indication extension
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.
Proprietary name	Crysvita
Manufacturer	Kyowa
Route of administration	Subcutaneous
Therapeutical formulation	Injection
Budgetting framework	Intermural (MSZ)
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 route	Centralised (EMA)
ATMP	No
Submission date	January 2021
Expected Registration	November 2021
Orphan drug	Yes
Registration phase	Registration application pending

Therapeutic value	No estimate possible yet

There is currently nothing known about the expected patient volume.

There is currently nothing known about the expected cost.

There is currently nothing known about the possible total cost.

There is currently nothing known about off label use.

There is currently nothing known about indication extensions.

There is currently no futher information available.