Eflornithine / sulindac

Active substance
Eflornithine / sulindac
Domain
Oncology and Hematology
Reason of inclusion in Horizonscan Geneesmiddelen
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.

1. Product

Manufacturer
Cancer Prevention Pharma
Mechanism of action
Combination therapy
Route of administration
Oral
Therapeutical formulation
Tablet
Budgetting framework
Intermural (MSZ)
Additional comments
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

2. Registration

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

3. Therapeutic value

Current treatment options
Poliepectomie
Therapeutic value
Potential equal value
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.
Duration of treatment
Not found
Frequency of administration
1 times a day
Dosage per administration
750 mg eflornithine en 150 mg sulindac
References
NCT01483144

4. 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 comments
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.

5. Expected cost per patient per year

There is currently nothing known about the expected cost.

6. Potential total cost per year

There is currently nothing known about the possible total cost.

7. Off label use

There is currently nothing known about off label use.

8. Indication extension

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

9. Other information

There is currently no futher information available.