Odevixibat Medicine 12 June 2019 Extended indication Treatment of Progressive Familial Intrahepatic Cholestasis Therapeutic value No judgement Total cost Registration phase Clinical trials Product Active substance Odevixibat Domain Metabolism and Endocrinology Reason of inclusion New medicine (specialité) Main indication Liver diseases Extended indication Treatment of Progressive Familial Intrahepatic Cholestasis Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Albireo Portfolio holder Mechanism of action Other Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Sodium-bile acid cotransporter-inhibitor. A4250 blokkeert galzuurresorptie in darm en onderbreekt de enterohepatische kringloop. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity New medicine with Priority Medicines (PRIME) ATMP Unknown Submission date 2021 Expected Registration 2022 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks 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 This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References www.orpha.net Additional remarks Expected patient volume per year Patient volume 1 - 4 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References www.orpha.net, CBS Additional remarks "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 Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References clinicaltrials.gov Additional remarks Er zijn geen fase II/III studies op andere indicatiegebieden op dit moment. Other information Additional remarks