Obeticholic Acid in Pediatric Subjects With Biliary Atresia

Key Inclusion Criteria:

1. Male or female pediatric subjects ≥2 to \<18 years old
2. Diagnosis of biliary atresia
3. Demonstrated successful HPE (also known as Kasai portoenterostomy) as defined by total bilirubin \<2 mg/dL (34.2 μmol/L) at least 3 months post-HPE procedure.
4. Able to swallow tablets (ie, tablet or mini-tablet formulation)

Key Exclusion Criteria:

1. Prior liver transplant or active status on transplant list
2. Conjugated (direct) bilirubin ≥ULN of site specific reference range
3. If conjugated bilirubin is not available: total bilirubin ≥2 mg/dL (34.2 μmol/L)
4. Platelets \<150,000/μL
5. INR ≥1.5
6. Current or history of complications of decompensated chronic liver disease including:

   1. high-risk gastroesophageal varices and/or variceal bleeding
   2. clinically evident ascites related to portal hypertension
   3. hepatic encephalopathy
   4. prior placement of portosystemic shunt
   5. hepatopulmonary syndrome or portopulmonary hypertension
   6. hepatorenal syndrome
7. Current intractable pruritus or requires systemic treatment for pruritus within 3 months of Screening (e.g., with bile acid sequestrants or rifampicin)
8. Height and weight Z-score \<-2 per site specific ranges
9. Acholic (pale) stools
10. AST \>4x ULN
11. ALT \>4x ULN
12. GGT \>500 U/L
13. Anticoagulation therapy
14. Albumin \<3.5 g/dL
15. Ongoing current cholangitis
16. Choledochal cystic disease
17. Renal disease defined as serum creatinine \>ULN for subject's age, prior to enrollment