Version 1

Eflornithine / sulindac

Medicine

Extended indication Delaying time to the first occurrence of any Familial Adenomatous Polyposis (FAP)-related event. This includes: 1) FAP related disease progression ind
Therapeutic value Possible equal value
Registration phase Clinical trials

Product

Active substance Eflornithine / sulindac
Domain Oncology and Hematology
Reason of inclusion New medicine (specialité)
Main indication Colon cancer
Extended indication Delaying time to the first occurrence of any Familial Adenomatous Polyposis (FAP)-related event. This includes: 1) FAP related disease progression indicating the need for excisional intervention involving the colon, rectum, pouch, duodenum and/or 2) clinically important events which includes progression to more advanced duodenal polyposis, cancer or death.
Manufacturer Cancer Prevention Pharma
Mechanism of action Combination therapy
Route of administration Oral
Therapeutical formulation Tablet
Budgetting framework Intermural (MSZ)
Additional remarks Eflornithine remt irreversibel het enzym ornithine decarboxylase, dat nodig is voor polyamine biosynthese, waardoor de vorming en groei van kankercellen wordt geremd. Sulindac, een NSAID, heeft potentieel antineoplastische activiteit

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Particularity New therapeutical formulation
ATMP Unknown
Submission date 2019
Expected Registration 2020
Orphan drug Yes
Registration phase Clinical trials

Therapeutic value

Current treatment options Poliepectomie
Therapeutic value Possible equal value

This assessment does not indicate any potential inclusion in the package.
Substantiation Nog geen studiedata bekend. Echter, bij patiënten is poliepectomie en eventueel een operatie zeer effectief en is overlevingskans van deze patiëntengroep gelijk aan gemiddelde bevolking.
Frequency of administration 1 times a day
Dosage per administration 750 mg eflornithine en 150 mg sulindac
References NCT01483144

Expected patient volume per year

Patient volume 500 - 1,700

Market share is generally not included unless otherwise stated.
References www.erfelijkheid.nl/, https://www.nhg.org/node/1957, https://www.sps.nhs.uk/medicines/eflornithine/
Additional remarks Erfelijkheid.nl en NHG: over de hele wereld hebben ongeveer 7 tot 10 op de 100.000 mensen FAP. SPS: de prevalentie wordt geschat op 3 tot 6 personen op de 100.000. Uitgaande van 3 - 10 personen per 100.000 komt dit neer op zo'n 500 tot 1700 personen in Nederland.

Expected cost per patient per year

Potential total cost per year

Off label use

Off label use Unknown

Indication extension

Indication extensions Yes
Indication extensions Fase 3 studie "colon cancer risk reduction"
References http://www.canprevent.com/pipeline/

Other information