Version 2

Eflornithine

Medicine

Extended indication Eflornithine monotherapy for preventive treatment of relapse in high risk neuroblastoma in infants and toddlers, children, adolescents and adults up t
Therapeutic value Possible added value
Total cost € 2,696,447.00
Registration phase No registration expected

Product

Active substance Eflornithine
Domain Oncology
Reason of inclusion Indication extension IND
Main indication Brain cancer
Extended indication Eflornithine monotherapy for preventive treatment of relapse in high risk neuroblastoma in infants and toddlers, children, adolescents and adults up to 21 years of age who have demonstrated at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy.
Proprietary name Iwilfin
Manufacturer Norgine
Mechanism of action Unknown
Route of administration Oral
Therapeutical formulation Tablet
Budgetting framework Intermural (MSZ)

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Particularity Unknown
ATMP No
Submission date January 2025
Orphan drug Yes
Registration phase No registration expected
Additional remarks Teruggetrokken in juli 2025.

Therapeutic value

Therapeutic value Possible added value

This assessment does not indicate any potential inclusion in the package.
Substantiation “The median OS with eflornithine plus lomustine was 34.9 months vs 23.5 months with lomustine alone (HR, 0.64; log-rank P = .016). Similarly, the median PFS of was 15.8 months vs 7.2 months with the combination vs lomustine, respectively (HR, 0.58; log-rank P = 0.015).” Er is sprake van enkele maanden verlenging van de overall en progressievrije overleving (3,4).
Frequency of administration 2 times a day
Dosage per administration 192 tot 768mg
References NCT02395666 (1); FDA prescribing information (2); Coleman et al. 2024 SNO Annual Meeting Presentation. (3); Expertopinie (4).
Additional remarks De aanbevolen IWILFIN-dosering, gebaseerd op het lichaamsoppervlak (m2): 1,5=768mg (vier tabletten) oraal tweemaal per dag. 0,75 tot 1,5=576mg (drie tabletten) oraal tweemaal per dag. 0,5 tot < 0,75=384mg (twee tabletten) oraal tweemaal per dag. 0,25 tot < 0,5=192mg (één tablet) oraal tweemaal per dag.

Expected patient volume per year

Patient volume 10 - 15

Market share is generally not included unless otherwise stated.
References NKR 2021 (1); Expertopinie (2).
Additional remarks In 2021 waren er 30 diagnoses neuroblastoom in Nederland (1). Volgens experts zouden er tien tot vijftien patiënten per jaar in aanmerking komen voor dit middel (2).

Expected cost per patient per year

Cost € < 207,419.00

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 Drugs.com (1)
Additional remarks In de Verenigde Staten kosten een tablet Iwilfin van 192mg $98.74 (omgerekend ongeveer €94,65) (1). Bij een toediening van zes tabletten per dag kost een behandeling €207.419,80 per patiënt per jaar (€94,65∗6∗365,24). De prijs in de Verenigde Staten is doorgaans hoger dan in Nederland.

Potential total cost per year

Total cost € 2,696,447.00

Off label use

Off label use Unknown

Indication extension

Indication extensions Unknown

Other information