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A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in South Korea

A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in South Korea

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Phase N/A

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Overview

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.

Eligibility

Inclusion criteria:

  1. 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
  2. On (or starting) active odevixibat treatment Note: Participants can remain in the registry during odevixibat treatment interruptions

Exclusion criteria:

  1. Currently participating in a clinical trial with odevixibat
  2. Currently participating in any interventional clinical trial for PFIC
  3. Have any contraindication to odevixibat as per the approved label in South Korea.

Study details
    Progressive Familial Intrahepatic Cholestasis

NCT07185919

Ipsen

1 February 2026

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