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Porcine recombinant factor VIII (Obizur; OBI ‐1; BAX 801): product characteristics and preclinical profile
Author(s) -
Lillicrap D.,
Schiviz A.,
Apostol C.,
Wojciechowski P.,
Horling F.,
Lai C. K.,
Piskernik C.,
Hoellriegl W.,
Lollar P.
Publication year - 2016
Publication title -
haemophilia
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.213
H-Index - 92
eISSN - 1365-2516
pISSN - 1351-8216
DOI - 10.1111/hae.12784
Subject(s) - immunogenicity , medicine , recombinant dna , pharmacology , pharmacokinetics , tolerability , drug , antibody , baby hamster kidney cell , adjuvant , immune system , immunology , virology , adverse effect , virus , biology , biochemistry , gene
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 (rp FVIII ), Obizur ( OBI ‐1; BAX 801), 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 rp FVIII 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.