An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia

This is a Phase 3, multi-center, open-label extension study to evaluate the long-term efficacy and safety of odevixibat in patients with Biliary Atresia (BA). Patients who completed treatment in the A4250-011 BOLD study and meet eligibility criteria for Study A4250-016 (BOLD-EXT) can participate. The duration of the treatment period is 104 weeks, followed by a 4-week Safety Follow-up Period. Patients who wish to continue receiving odevixibat after 104 weeks can remain on treatment in the optional extension period (OEP).

Up to 180 patients will be enrolled at approximately 70 sites in the North America, Europe, the Middle East and Asia-Pacific.

Inclusion Criteria:

• Completion of the 104-week Treatment Period of Study A4250-011
• Signed informed consent by caregiver

Exclusion Criteria:

• Patients who were not compliant with study drug treatment or procedures in Study A4250-011 as per the investigator's discretion
• Any conditions or abnormalities which, in the opinion of the investigator, may compromise the safety of the patient, or interfere with the patient participating in or completing the study
• Known hypersensitivity to any components of odevixibat
• Patients who are scheduled for a liver transplant or are likely to require a liver transplant in the immediate future based on the investigator's judgment

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications.

Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.

Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and/or EU.

Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).