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Recombinant activated factor VII (rFVIIa): characterization, manufacturing, and clinical development
B Jurlander1, L Thim, N K Klausen
1IO Biopharm, Novo Nordisk A/S, Novo Allé, DK-2880 Bagsvaerd, Lyngby, Denmark. bju@novonordisk.com
Seminars in Thrombosis and Hemostasis
|September 8, 2001
Summary
Recombinant activated factor VII (rFVIIa) effectively treats bleeding in hemophilia patients with inhibitors. This therapy, identical in sequence to plasma-derived FVIIa, shows promise for other coagulation disorders.
Area of Science:
- Biochemistry
- Hematology
- Pharmacology
Background:
- Recombinant activated factor VII (rFVIIa) is a treatment for hemophilia patients with inhibitors.
- rFVIIa initiates coagulation by binding to tissue factor, promoting thrombin generation.
- It has been used successfully in other bleeding disorders with impaired thrombin generation.
Purpose of the Study:
- To review the mechanism of action, characterization, manufacturing, and clinical evidence of rFVIIa.
- To highlight its efficacy and safety in treating various bleeding conditions.
Main Methods:
- The review synthesizes preclinical and clinical data on rFVIIa.
- Manufacturing process involves expression in baby hamster kidney cells and purification.
- Characterization confirms identical amino acid sequence and qualitatively identical posttranslational modifications to plasma-derived FVIIa.
Main Results:
- rFVIIa activity is indistinguishable from plasma-derived FVIIa (pdFVIIa).
- Manufacturing ensures complete autoactivation of recombinant factor VII to rFVIIa.
- Clinical registries are collecting data on its use in various bleeding disorders.
Conclusions:
- rFVIIa is a well-characterized therapeutic agent with proven efficacy.
- Its manufacturing process ensures a high-quality product.
- Ongoing data collection will further define its role in managing bleeding disorders.