This study will collect information from people with Progressive Familial Intrahepatic Cholestasis (PFIC) as they use odevixibat in their daily lives. Odevixibat is a medicine that helps people with PFIC, a type of rare disease that makes their liver not work well and causes itching and yellow skin. Odevixibat was first allowed to be used for PFIC in babies older than 6 months by the European Medicines Agency (EMA) on 16 July 2021 and by the United States Food and Drug Administration (FDA) on 20 July 2021 for itching in babies older than 3 months. Obevixibat was approved by the Ministry of Food and Drug Safety (MFDS) in South Korea on 23 August 2024.
This study will collect information to see how well and how safe odevixibat is in the long run for participants in South Korea.
Diagnosed with PFIC (all types) who have been prescribed odevixibat (independently of the decision to enrol the participant in this registry) by their treating physician
On (or starting) active odevixibat treatment Note: Participants can remain in the registry during odevixibat treatment interruptions
Currently participating in a clinical trial with odevixibat
Currently participating in any interventional clinical trial for PFIC
Have any contraindication to odevixibat as per the approved label in South Korea.
The registry population will comprise participants with PFIC (all types) enrolled into the Ipsen odevixibat PFIC registry. Participants with PFIC who have been prescribed odevixibat by their treating physician will be eligible.
Other
Prospective
Not listed
Percentage of participants experiencing Adverse Events (AEs)
An Adverse event (AE) is any untoward medical occurrence, temporally associated with the use of study intervention, whether or not related to the study intervention.
Event-free survival (EFS)
EFS is defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion, liver transplant, or death
Surgical biliary diversion-free survival
Surgical biliary diversion-free survival is defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death
Liver transplant-free survival
Liver transplant-free survival is defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death
Overall survival (OS)
OS is defined as the time from the start of odevixibat treatment to death
