Journal
HAEMOPHILIA
Volume 22, Issue 2, Pages 308-317Publisher
WILEY
DOI: 10.1111/hae.12784
Keywords
acquired haemophilia A; bypass agents; FVIII; FVIII inhibitors; Obizur; recombinant porcine FVIII
Categories
Funding
- National Institutes of Health [U54 HL112309]
- Hemophilia of Georgia, Inc.
- Baxalta US Inc.
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Introduction: Acquired haemophilia A (AHA) is a rare, often severe, auto-immune bleeding disorder caused by the development of inhibitory antibodies (inhibitors) to factor VIII (FVIII). Bypassing agents, recombinant activated FVII or activated prothrombin complex concentrate, are currently recommended as first-line treatments to control bleeding events in patients with AHA. Aim: A plasma-derived porcine FVIII (Hyate: C, Ipsen, UK) was used as a first-line treatment for AHA but was discontinued in 2004 due to viral safety concerns. A recombinant pFVIII (rpFVIII), Obizur (OBI-1; BAX801), which is expected to have a similar efficacy profile to Hyate: C but with a superior safety profile was developed and recently approved by the US Food and Drug Administration for the treatment of AHA. Methods: Obizur manufacturing begins with the expression of B domain deleted rpFVIII by genetically modified baby hamster kidney-derived cells. The final purified and lyophilized drug product has a negligible risk of viral contamination and contains no animal-derived plasma proteins. Obizur was evaluated for immunogenicity, tolerability, pharmacokinetics and bleeding times in preclinical models including in haemophiliac dogs, cynomolgus monkeys and FVIII-knockout mice. Results: Preclinical animal studies show that the efficacy and immunogenicity of Obizur are similar to that of Hyate: C and that Obizur has a more favourable safety profile. Conclusions: Obizur is a highly purified recombinant porcine FVIII drug product that has been demonstrated to have a favourable safety and efficacy profile when compared with Hyate: C and can be a valuable treatment option for control of bleeding in AHA patients.
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