Challenges for new haemophilia products from a manufacturer's perspective

摘要

The development of new and improved therapeutic options for the management of haemophilia is a great challenge for both physicians and manufacturers. After factor VIII concentrates became widely available, progress in medicine and advances in biotechnology led to the development of virus-inactivated, plasma-derived products of high purity and recombinant products, which are currently further improved regarding an extended half-life, potentially allowing for effective prophylaxis with reduced dosing frequency and hopefully less immunogenicity. This article describes some of the challenges that were experienced by the manufacturer during the development of the high-purity, plasma-derived factor VIII concentrate, Beriate~R P, which, after implementation of a nanofiltration step in its manufacture, is now designated as Beriate~R; it also outlines the challenges and achievements to date with half-life extended products such as the recombinant fusion proteins linking coagulation factor Vila (rVIIa-FP) and factor IX (rlX-FP) with albumin.