This registry-based study will collect information from people with Progressive Familial Intrahepatic Cholestasis (PFIC) who take odevixibat (Bylvay) as part of routine clinical care in China.
PFIC is a rare genetic liver disease that affects bile secretion and can cause bile acids to build up in the liver, which may lead to symptoms such as severe itching (pruritus).
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. Odevixibat is approved for the treatment of pruritus in PFIC and was approved in China on 01 December 2024 for patients 6 months of age and older with PFIC.
The main aim of this registry is to assess long-term real-world safety (based on adverse events) and to describe effectiveness outcomes.
Diagnosed with PFIC (all types) who have been prescribed odevixibat (independently of the decision to enroll the participant in this registry) by their treating physician
On (or starting) active odevixibat treatment (participants can remain in the registry during odevixibat treatment interruptions)
Signed informed consent and assent, as appropriate
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 China
Participants with Progressive Familial Intrahepatic Cholestasis (PFIC) (all types) who have been prescribed odevixibat by their treating physician will be eligible. Participants who started odevixibat treatment before the implementation of the registry may also be enrolled.
Other
Prospective
Not listed
Percentage of participants experiencing adverse events (AEs)
An AE is any untoward medical occurrence in a participant administered a medicinal product and does not necessarily have a causal relationship with treatment
Percentage of participants experiencing serious adverse events (SAEs)
SAEs are collected as part of safety reporting and include events meeting seriousness criteria (e.g., death, life-threatening, hospitalization, etc.) as defined in the protocol
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
Defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death.
Liver transplant-free survival
Defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death.
Overall survival
Defined as time from the start of odevixibat treatment to death.
