Biopharmaceuticals: Approval Trends in 2008

Ten biopharmaceuticals gained marketing authorization in the US or EU in 2008, although only four were new approvals.
Oct 01, 2009
Volume 22, Issue 10

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The year 2008 was, like 2007, a somewhat disappointing year in terms of the number of new biopharmaceuticals approved. The year witnessed the approval of eight recombinant proteins or engineered antibody-based products in the US, and two such biopharmaceuticals in the European Union (EU, Table 1). These products included hormones, interferons, blood factors, and fusion proteins—two each—as well as one antibody-based product and a colony-stimulating factor.

Although 10 approvals does not appear an unduly low number, only four of those products contained genuinely new active ingredients (Arcalyst, Cimzia, Nplate, and Recothrom; Table 1). Arcalyst and Nplate are fusion proteins, Cimzia is an antibody Fab', and Recothrom is a recombinant thrombin (blood coagulation factor IIa).

Biosimilar Products
The other six (Accretropin, Extavia, Filgrastim ratiopharm, NovoLog mix, PEGintron/ribetol combo, and Xyntha) are effectively reformulated, biosimilar or "me too" versions of previously approved products (see sidebar: Biosimilar Products).

Product indications too are somewhat diverse and none aim to treat cancer. Also notable amongst the 2008 crop is the paucity of antibody-based products. No full-length antibody came to the market last year, although one antigen-binding antibody fragment (Cimzia) did gain approval.

In terms of expression systems used, the 2008 approvals also appeared to buck the general trend toward mammalian-based systems. Six of the 10 approved products are expressed in E. coli, one in S. cerevisiae, and three in Chinese hamster ovary (CHO) cell lines. However, the E. coli bias is less obvious in the context of genuinely novel active pharmaceutical ingredients approved, of which two are expressed in E. coli and two in CHO cells.

In most previous years too, some overlap in EU and US approvals was evident.1–4 Not so in 2008. Although approval numbers in the US were modest, they were even more disappointing in EU, with only two products coming on the market. Moreover, neither of the EU approvals (Extivia and Filgrastim Ratiopharm) were genuinely novel (see sidebar).

Table 1. Biopharmaceuticals (defined as recombinant proteins, monoclonal antibody, and nucleic acid-based products) approved in the US or EU in 2008
In financial terms too, the majority of products approved, are expected to generate modest as opposed to blockbuster-level revenues. Nplate, Xyntha, and Recthrom sales are each projected to reach the $300–500 million mark.5 Arcalyst, aimed at treating CAPS (Table 1) has designated orphan status. With an estimated total US population base of only 300–500 patients, the market potential has been estimated at $10–35 million, despite the drug's premium price (approximately €250,000 per patient annually).6

The only product with a likely potential of reaching blockbuster status is that of Cimzia.5 Although initially approved only for the treatment of Chron's disease, it was subsequently approved (May 2009) for the treatment of rheumatoid arthritis, greatly increasing its revenue scope.

Overall, therefore, 2008 was a modest year in terms of biopharmaceutical approvals. Analysts, however, are hopeful that this will represent a low point, with approvals likely to steadily increase once again in the coming years.5

Individual monographs for products are presented below. Monograph for Extavia, Novologmix 50:50, and PEGintron ribetol are not provided, given their particularly close similarity to well-established, previously approved products.

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