Evaluation of Avatrombopag for the Treatment of Thrombocytopenia in Japanese Adults With Chronic ITP

Inclusion Criteria:

• Subject has a confirmed diagnosis of chronic immune thrombocytopenia (ITP) (≥12months duration) and has had an insufficient response to a previous ITP treatment,in the opinion of the Investigator.

• Subject has an average of 2 platelet counts <30×10^9/L (no single count can be >35×10^9/L). The 2 samples must be obtained ≥48 hours and ≤2 weeks apart.

Exclusion Criteria:

• Subjects with known secondary immune thrombocytopenia (e.g., with known Helicobacterpylori-induced ITP, subjects infected with known human immunodeficiency virus (HIV)or hepatitis C virus (HCV) or subjects with known systemic lupus erythematosus).

• Subjects with known inherited thrombocytopenia (e.g., Myosin Heavy Chain 9 (MYH-9)disorders) or hereditary thrombophilic disorders (e.g., Factor V Leiden,antithrombin III deficiency).

• History of myelodysplastic syndrome (MDS).

• History of arterial or venous thrombosis.

• Subjects with a history of significant cardiovascular disease (e.g., congestiveheart failure (CHF) New York Heart Association Grade III/IV, arrhythmia known toincrease the risk of thromboembolic events [e.g., atrial fibrillation], angina,coronary artery stent placement, angioplasty, coronary artery bypass grafting).

• Subjects with a history of cirrhosis, portal hypertension, or chronic activehepatitis.

• Subjects with concurrent malignant disease or receiving cytotoxic chemotherapy for areason other than ITP treatment.

• Use of immunoglobulins (IVIg and anti-D) or corticosteroid rescue therapy within 1week of Day 1/Baseline.

• Splenectomy or use of rituximab within 12 weeks of Day 1/Baseline.

• Use of romiplostim or eltrombopag within 1 week of Day 1/Baseline.

• Use of chronic corticosteroid treatment or azathioprine within 4 weeks of Day 1/Baseline, unless receiving a stable dose for at least 4 weeks.

• Use of mycophenolate mofetil, cyclosporin A, or danazol within 4 weeks of Day 1/Baseline, unless receiving a stable dose for at least 12 weeks.

• Use of cyclophosphamide or vinca alkaloid regimens within 4 weeks of Baseline Visit.

• Currently receiving moderate or strong dual inhibitors/inducers of CYP2C9 andCYP3A4.

• Serum creatinine ≥1.5× the upper limit of normal (ULN).

• Serum bilirubin ≥2×ULN.

• Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥3×ULN.

• Females who are pregnant (positive beta-human chorionic gonadotropin (β-hCG) test)or breastfeeding.

• Received treatment with an investigational drug within 30 days or 5 half-lives (whichever is longer) before Day 1/Baseline.