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Biotechnology products and indications I. Proteins
Published in Ronald P. Evens, Biotechnology, 2020
The coagulation proteins number 30, as listed in Table 8.6. Six companies market most of the products worldwide, that is, Bayer, CSL Behring, Novo Nordisk, Octapharma, Pfizer, and Takeda. Blood factors are very large proteins (thousands of amino acids in two chains [- A & B ]) and are involved in normal blood coagulation as cofactors in the coagulation cascade [Factor 7, F.8, F.9, F.10, F.13, and thrombin, plus von Willebrand factor]. The deficiency of any one blood factor, often a genetic deficiency, leads to serious bleeding disorders (hemophilia), but it is fully correctable through replacement therapy with these rDNA proteins. The factors are recombinant DNA proteins employing two possible hamster host cells, baby kidney (BKH) or Chinese ovaries (CHO). Factor 8 is available in 18 products, all with the same indication and use, and Factor 9 in 6 products. The original blood factors were short acting, but the use of pegylation (Adynovate, Esperoct, Jivi, Rebinym) and fusion proteins (e.g., Afystyla, Alprolix) extends the products’ half-lives substantially. Also, further molecular engineering of the protein domains, mostly B-domain deletion or truncation, creates additional molecules and products; however, the indications are the same as for the originator product. Other coagulation proteins are marketed for Factor 7 and von Willebrand deficiencies. These recombinant protein blood factors replace natural blood derivative products and avoid the potential viral contamination and immune reactions that were previously observed in these patients receiving blood derived products. Additionally, both a thrombin and antithrombin protein are marketed. An antidote to blood thinners is available as well, Andexxa. One coagulation protein (Atryn) is an antithrombin that involves manufacture in transgenic goats to produce the protein in their milk. Thrombocytopenia purpura has a protein therapeutic available (Nplate).
Eptacog beta: a novel recombinant human factor VIIa for the treatment of hemophilia A and B with inhibitors
Published in Expert Review of Hematology, 2019
Christine Biron-Andreani, Jean-François Schved
Today, two therapeutic proteins isolated from the milk of transgenic animals, C1 inhibitor (Ruconest®, Pharming, Leiden, The Nederlands) and antithrombin (ATryn®, rEVO Biologics, Framingham, MA, USA), have obtained their marketing authorization both in the United States and Europe.