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

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.

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

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

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.

Expected cost per patient per year

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

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

Other information

There is currently no futher information available.