Sodium thiosulfate

Extended indication	Pedmarqsi is indicated for the prevention of ototoxicity induced by cisplatin chemotherapy in patients 1 month to <18 years of age with localised, non-metastatic, solid tumours.
Therapeutic value	No estimate possible yet
Total cost	4,050,000.00
Registration phase	Registered

Product

Active substance	Sodium thiosulfate
Domain	Oncology
Reason of inclusion	New medicine (specialité)
Main indication	Oncology other
Extended indication	Pedmarqsi is indicated for the prevention of ototoxicity induced by cisplatin chemotherapy in patients 1 month to <18 years of age with localised, non-metastatic, solid tumours.
Proprietary name	Pedmarqsi
Manufacturer	Fennec Pharmaceuticals
Mechanism of action	Antioxidant
Route of administration	Intravenous
Therapeutical formulation	Intravenous drip
Budgetting framework	Intermural (MSZ)

Registration

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
ATMP	No
Submission date	March 2020
Expected Registration	May 2023
Orphan drug	No
Registration phase	Registered
Additional remarks	Positieve CHMP opinie 30 maart 2023

Therapeutic value

Therapeutic value	No estimate possible yet
Substantiation	De effectiviteit van sodium thiosulfaat op het voorkomen van ototoxiciteit is aangetoond middels onderzoek. Daarentegen is ook aangetoond dat sodium thiosulfaat de effectiviteit van cisplatine mogelijk teniet doet (Freyer, 2014). De algehele survival van de groep patiënten die sodium thiosulfaat is namelijk kleiner dan dat van de controle groep.
Dosage per administration	16 g/m2
References	NCT00716976, Freyer, D. (2014). The effects of sodium thiosulfate (STS) on cisplatin-induced hearing loss: A report from the Children’s Oncology Group. Journal Of Clinical Oncology, 32(15_suppl), 10017-10017.
Additional remarks	Dosering per toediening: 16g/m2 or 533mg per kg for patients whose therapeutic protocol administers cisplatin on a per kg basis due to young age or small body).

Expected patient volume per year

Patient volume	50 - 100 Market share is generally not included unless otherwise stated.
References	Expert opinie(1);
Additional remarks	Op basis van het aantal patiënten van 1 tot 18 jaar dat cisplatin ontvangt is de verwachting dat er ongeveer 50 tot 100 patiënten in aanmerking zullen komen (1).

Patient volume

50 - 100

Market share is generally not included unless otherwise stated.

References

Expert opinie(1);

Additional remarks

Op basis van het aantal patiënten van 1 tot 18 jaar dat cisplatin ontvangt is de verwachting dat er ongeveer 50 tot 100 patiënten in aanmerking zullen komen (1).

Expected cost per patient per year

Cost	54,000.00
References	https://dawsonjames.com/
Additional remarks	In een equity onderzoek van DawsonJames securities wordt een prijs van $60,000 genoemd, ongeveer €54.000.

Potential total cost per year

Total cost	4,050,000.00 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.

Total cost

4,050,000.00

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.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension	No
References	adisinsight

Other information

There is currently no futher information available.