In terms of product approvals, the past year can be considered reasonably successful. Twelve biopharmaceutical marketing applications were approved in the United States and/or the European Union in 2012 (see Table I). Approval numbers for the past year are in line with those recorded over the previous five years (1-4), which reveal a statistical average of 12.2 approvals annually (see Figure 1).
Figure 1: Biopharmaceutical approval numbers in European Union and/or United States over the past five years.
Products approved included three enzymes, three antibodies, and two fusion proteins, as well as a gene therapy product, a regulatory peptide, a blood product, and a colony stimulating factor. Three products are indicated against cancer and a further two against genetic diseases, while the remainder target a wide range of medical conditions.
In terms of expression systems used, 5 of the 11 protein-based products approved are produced using mammalian (mainly CHO) cell lines, three are expressed in E. coli, two in yeast-based systems (one each in S. cerevisiae and P. pastoris), and one product is produced in an engineered plant-based system. These figures once again reflect the ongoing dominance of mammalian-based expression systems in terms of biopharmaceutical manufacture.
Digging a little deeper reveals that the majority of approvals (10 of the 12) were first-time approvals in 2012. Two of the products (Adcetris and Eylea), although newly approved in one region last year, had gained approval in 2011 in the other region (Table I). Elelyso, moreover, is not the first glucocerebrosidase approved, nor is Tbo-filgrastim the first G-CSF-based product to come on the market.
Table I: Biopharmaceuticals (recombinant proteins, monoclonal antibody, or nucleic acid-based products) approved in the USA and/or European Union in 2012. Abbreviations: r = recombinant; rh = recombinant human; VEGF = vascular endothelial growth factor; AAV1 = adeno-associated virus, serotype 1; G-CSF = granulocyte colony stimulating factor; LPL = lipoprotein lipase.
Several of the newly approved products are particularly noteworthy in terms of technological or medical innovation, and it is those products that are discussed in most detail below. As Adcetris and Eylea were included in last year's review (1), they are not considered further herein.