Burosumab

Extended indication	X-linked hypophosphatemia
Therapeutic value	Possible added value

Product

Active substance	Burosumab
Domain	Metabolism and Endocrinology
Main indication	Metabolic diseases
Extended indication	X-linked hypophosphatemia
Proprietary name	Crysvita
Manufacturer	Kyowa
Route of administration	Subcutaneous
Therapeutical formulation	Injection
Budgetting framework	Extramural (GVS)
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 route	Centralised (EMA)
Type of trajectory	Normal trajectory
Submission date	January 2017
Expected Registration	February 2018
Orphan drug	Yes
Additional remarks	Positieve opinie CHMP december 2017.

Therapeutic value	Possible added value
Substantiation	Met name bij kinderen, ten opzichte van fosfaatsuppletie en calcitriol.
Duration of treatment	Not found

Patient volume	< 850 Market share is generally not included unless otherwise stated.
Additional remarks	Orphanet: prevalence of approximately 1/ 20,000. Dit betekent 850 per 17miljoen inwoners.

Patient volume

< 850

Market share is generally not included unless otherwise stated.

Additional remarks

Orphanet: prevalence of approximately 1/ 20,000. Dit betekent 850 per 17miljoen inwoners.

Additional remarks	Waarschijnlijk hoge kosten

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.