Recombinant B domain deleted porcine factor VIII for the treatment of bleeding episodes in adults with acquired hemophilia A.
Hemophilia A is an inherited deficiency of clotting factor VIII (FVIII) often complicated by inhibitor development (CHAWI) in which neutralizing antibodies block the therapeutic benefit of replacement therapy. Inhibitors to FVIII can also be seen in an auto-immune disease known as acquired hemophilia A (AHA). 'Bypassing' therapies have been shown to provide hemostasis but dosing must be done empirically because current assays cannot measure objective markers of treatment efficacy and safety. A recombinant porcine sequence factor VIII (r-pFVIII) has been developed for the management of AHA. Preclinical, Phase I and Phase II clinical research studies in CHAWI subjects showed therapeutic potential and safety of this agent. A Phase II/III study in AHA with serious bleeding episodes shows a positive response in all subjects after administration. Based on current preclinical and clinical trial data, r-pFVIII should become the first line of treatment in the management of hemorrhage in patients with AHA.
Division of Hematology-Oncology, Children's Hospital of Los Angeles, 4650 Sunset Boulevard, Los Angeles, CA 90027, USA.
Edward
E
Gomperts
D000328:Adult; D000818:Animals; D017321:Clinical Trials, Phase I as Topic; D017322:Clinical Trials, Phase II as Topic; D017326:Clinical Trials, Phase III as Topic; D015944:Factor VIIIa / Q000494:pharmacology / Q000627:therapeutic use*; D006467:Hemophilia A / Q000175:diagnosis / Q000188:drug therapy*; D006801:Humans; D011994:Recombinant Proteins / Q000494:pharmacology / Q000627:therapeutic use*; D012720:Severity of Illness Index; D013552:Swine; D016896:Treatment Outcome
D011994:Recombinant Proteins; D015944:Factor VIIIa
OBIZUR; acquired hemophilia A; factor VIII; hemophilia A; inhibitor; recombinant porcine FVIII