Version 8

Sodium thiosulfate

Medicine

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,
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
Particularity Unknown
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

This assessment does not indicate any potential inclusion in the package.
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).

Expected cost per patient per year

Cost € 54,000.00

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 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

Off label use

Off label use Unknown

Indication extension

Indication extensions No
References adisinsight

Other information