INTERCEPT Blood System for Plasma
Safety and Efficacy Data: Clinical Use

Congenital Coagulation Factor Deficiencies
A single-arm, open-label clinical trial was conducted to evaluate efficacy and safety of INTERCEPT Plasma in patients with congenital deficiencies of coagulation factors I (fibrinogen), II, V, VII, X, XI, and XIII as well as protein C.  The results of this 34 patient  trial demonstrated that, for most factors evaluated, INTERCEPT Plasma provided coagulation factor recovery and pharmacokinetics comparable to conventional plasma, as reported in the literature, and PT and aPTT responses sufficient for adequate hemostasis.  The respective terminal half-lives and clearances for patients with deficiencies of coagulation factors V, VII, X, XI and protein C were comparable to literature references.  Terminal half-life results for factors I, II and XIII were low relative to the medical literature.  These results may have been due to the very small number of patients evaluated (n of 1-3 for each factor) and differences in the methods of analysis.  Hemostasis was achieved for all therapeutic transfusions and INTERCEPT Plasma was well-tolerated.

Acquired Coagulation Factor Deficiencies
A randomized, controlled, double-blinded clinical trial was conducted to evaluate efficacy and safety of INTERCEPT Plasma compared to conventional fresh frozen plasma in patients with acquired coagulation deficiencies.  The results of this 121 patient clinical trial demonstrated the efficacy of INTERCEPT Plasma for treatment of coagulopathy resulting from chronic liver disease, including a significant proportion of patients undergoing orthotopic liver transplantation.  Maintenance of adequate hemostasis during orthotopic liver transplantation and other invasive procedures was similar between treatment groups.  There were no significant differences in adverse events, including hepatic artery thrombosis, deaths, or transfusion reactions between patients treated with INTERCEPT Plasma and those treated with conventional fresh frozen plasma.

Therapeutic Plasma Exchange
A randomized, controlled, double-blinded clinical trial was conducted to evaluate efficacy and safety of INTERCEPT Plasma compared to conventional fresh frozen plasma for therapeutic plasma exchange in patients with thrombotic thrombocytopenic purpura (TTP).  The results of this 35 patient clinical trial demonstrated that therapeutic response to plasma exchange with INTERCEPT Plasma was not different than response to conventional fresh frozen plasma in terms of both rates of TTP remission and relapse, and time to remission and relapse.  As patients received daily plasma volume exchanges over one or two 35-day cycles of exchange, the exposure to INTERCEPT Plasma in this study represents a 10-fold higher exposure when compared to transfusion studies where patients were treated for congenital or acquired coagulopathies.  The safety profile of INTERCEPT Plasma in this setting was similar to conventional fresh frozen plasma.  No evidence of antibody formation to amotosalen neoantigens was observed.

Technical Data Sheets section page 7 of 7