Odevixibat

Active substance
Odevixibat
Domain
Metabolism and Endocrinology
Reason of inclusion in Horizonscan Geneesmiddelen
New medicine (specialité)
Main indication
Liver diseases
Extended indication
Treatment of Progressive Familial Intrahepatic Cholestasis

1. Product

Manufacturer
Albireo
Mechanism of action
Other
Route of administration
Oral
Therapeutical formulation
Capsule
Additional comments
Sodium-bile acid cotransporter-inhibitor. A4250 blokkeert galzuurresorptie in darm en onderbreekt de enterohepatische kringloop.

2. Registration

Registration route
Centralised (EMA)
Particularity
New medicine with Priority Medicines (PRIME)
Submission date
2020
Expected Registration
2021
Orphan drug
Yes
Registration phase
Clinical trials

3. Therapeutic value

Current treatment options
Ursodeoxycholzuur (UDCA) -therapie moet bij alle patiënten worden gestart om leverschade te voorkomen. Bij sommige PFIC1- of PFIC2-patiënten kan galafleiding ook de pruritus verlichten en de progressie van de ziekte vertragen. De meeste PFIC-patiënten zijn uiteindelijk echter kandidaten voor levertransplantatie. Monitoring van hepatocellulair carcinoom, vooral bij PFIC2-patiënten, moet vanaf het eerste levensjaar worden aangeboden.
Therapeutic value
No judgement
Duration of treatment
Not found
References
www.orpha.net

4. Expected patient volume per year

Patient volume

1 - 4

Market share is generally not included unless otherwise stated.

References
www.orpha.net, CBS
Additional comments
"The exact prevalence remains unknown, but the estimated prevalence at birth varies between 1/50,000 and 1/100,000." Er zijn in 2017 169.836 kinderen geboren in Nederland (CBS), dit zou gaan om 1-4 personen.

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
No
References
clinicaltrials.gov
Additional comments
Er zijn geen fase II/III studies op andere indicatiegebieden op dit moment.

9. Other information

There is currently no futher information available.