Zolbetuximab

Extended indication	The treatment of adult patients with von Hippel-Lindau disease who require therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET), and for whom localized procedures are unsuitable or undesirable.
Therapeutic value	No estimate possible yet
Total cost	4,312,500.00
Registration phase	Registration application pending

Product

Active substance	Belzutifan
Domain	Oncology
Reason of inclusion	New medicine (specialité)
Extended indication	The treatment of adult patients with von Hippel-Lindau disease who require therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET), and for whom localized procedures are unsuitable or undesirable.
Manufacturer	MSD
Portfolio holder	MSD
Mechanism of action	Other
Route of administration	Oral
Therapeutical formulation	Tablet
Budgetting framework	Intermural (MSZ)
Additional remarks	Mode of action: HIF-2α inhibitor

Registration

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
ATMP	No
Submission date	December 2023
Expected Registration	January 2025
Orphan drug	No
Registration phase	Registration application pending

Therapeutic value

Therapeutic value	No estimate possible yet
Substantiation	De behandeling van deze kleine groep patiënten lijkt op de behandeling van andere patiënten met een gemetastaseerd niercarcinoom. Verdere resultaten worden nog afgewacht.

Expected patient volume per year

Patient volume	10 - 15 Market share is generally not included unless otherwise stated.
Additional remarks	based on clinical VHL experts input

Patient volume

10 - 15

Market share is generally not included unless otherwise stated.

Additional remarks

based on clinical VHL experts input

Expected cost per patient per year

Cost	< 345,000.00
References	Drugs.com
Additional remarks	In de VS kost belzutifan $31,243voor 90 tableten, wat neerkomt op een maand verbruik. Per jaar zou dat omgerekend ongeveer €345.000 kosten. De kosten in de EU zullen waarschijnlijk lager uitvallen.

Cost

< 345,000.00

References

Drugs.com

Additional remarks

In de VS kost belzutifan $31,243voor 90 tableten, wat neerkomt op een maand verbruik. Per jaar zou dat omgerekend ongeveer €345.000 kosten. De kosten in de EU zullen waarschijnlijk lager uitvallen.

Potential total cost per year

Total cost	4,312,500.00 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.

Total cost

4,312,500.00

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.

Off label use

Off label use	No

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.

Belzutifan