A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Patients With Inhibitors to Factor VIII or IX

Inclusion Criteria:

- be male with a diagnosis of congenital hemophilia A and/or B of any severity

- have one of the following:

- a positive inhibitor test Bethesda Unit (BU) ≥ 5 (as confirmed at screening by the
institutional lab), OR

- a BU<5 but expected to have a high anamnestic response to FVIII or FIX, as
demonstrated from the subject's medical history, precluding the use of Factor VIII or
IX products to treat bleedings, OR

- a BU<5 but expected to be refractory to increased dosing of FVIII or FIX, as
demonstrated from the subject's medical history, precluding the use of Factor VIII or
IX products to treat bleedings

- be 12 years or older, up to and including 75 years of age (NOTE: different age
restrictions may apply per local regulation and/or ethical considerations)

- have at least 3 bleeding episodes of any severity in the past 6 months be capable of
understanding and willing to comply with the conditions of the protocol

- have read, understood and provided written informed consent (patient and/or
parent(s)/legal guardian(s) if <18 years of age)

Exclusion Criteria:

- have any coagulation disorder other than hemophilia A or B

- be immuno-suppressed (i.e., the patient should not be receiving systemic
immunosuppressive medication, cluster of differentiation 4 (CD4) counts at screening
should be >200/µl)

- have a known allergy or hypersensitivity to rabbits

- have platelet count <100,000/mL

- have had within one month prior to first administration of the study drug in this
study a major surgical procedure (e.g. orthopedic, abdominal)

- have received an investigational drug within 30 days of the first study drug
administration, or is expected to receive such drug during participation in this
study

- have a clinically relevant hepatic (AST and/or alanine aminotransferase (ALT) >3
times the upper limit of normal) and/or renal impairment (creatinine >2 times the
upper limit of normal)

- have a history of arterial and/or venous thromboembolic events (such as myocardial
infarction, ischemic strokes, transient ischemic attacks, deep venous thrombosis or
pulmonary embolism) within 2 years prior to first dose of study drug, or current New
York Heart Association (NYHA) functional classification score of stage II -IV

- have an active malignancy (those with non-melanoma skin cancer are allowed)

- have any life-threatening disease or other disease or condition which, according to
the investigator's judgment, could imply a potential hazard to the patient, interfere
with the trial participation or trial outcome (e.g., a history of non-responsiveness
to bypassing products).