Abstract

The past decade has seen an explosion in the number of therapeutic proteins available for a wide spectrum of diseases. Some of these proteins are obtained from human plasma. Examples of these therapeutic proteins are albumin, intravenous immunoglobulins and prothrombin complex concentrates. The majority of new therapeutic proteins are, however, derived via recombinant DNA technology. There are other examples where the first therapeutic preparation was a crude preparation derived from plasma or tissue and where subsequent development has resulted in a recombinant form of the therapeutic protein. This article focuses on the development of therapeutics for the treatment of haemophilia A (deficiency of Factor VIII activity). The progression from crude plasma fractions to monoclonal-purified preparations to the more recent development of therapeutic concentrates via recombinant DNA technology is described in some detail. Finally, the current status of gene therapy for haemophilia A is evaluated. Both technical issues as well as market forces are described, as both have had significant impact on the product-development process.