Recombinant Protein Therapeutics

F E AT U R E

© 2004 Nature Publishing Group http://www.nature.com/naturebiotechnology

Recombinant protein therapeutics—success rates, market trends and values to 2010
Alex K Pavlou & Janice M Reichert
Despite entering its maturation phase, the recombinant protein sector will continue to drive the growth of medical biotechnology over the next six years.

Biotechnology, founded on theprinciples of recombinant DNA (rDNA) protein production, is and will probably continue to be an R&D-intensive sector. Beginning in the early 1970s, advances in molecular biology and genetic engineering have led to enormous progress in the ability to understand the biomolecular roots of human disease. Stanford University’s Paul Berg, the 1980 Nobel Prize winner in chemistry, first produced rDNA in1972. His lead was followed by a team led by Herbert Boyer (University of California, San Francisco), who in 1973 transformed Escherichia coli cells with recombinant plasmid and later founded Genetic Engineering Technology (Genentech, S. San Francisco, CA, USA). Boyer’s team perfected their methods, then developed and later launched, with the help of their licensing partner Eli Lilly (Indianapolis,IN, USA), the first human recombinant protein, recombinant human insulin (Humulin) in 1982. After more than two decades of continuous global expansion, business formation and technological diversification, the rDNA therapeutics sector now represents the core of the human medical biotechnology industry, worth over $32 billion in 2003. The rDNA therapeutics sector includes more than 110 companiesthat are involved in the discovery, development and marketing of rDNA products. These companies have a pipeline of more than 80 therapeutics in clinical development and a combined
Alex K Pavlou is at Datamonitor plc., Charles House 108-110 Finchley Road, London, NW3 5JJ (apavlou@datamonitor.com). Janice M Reichert is at Tufts Center for the Study of Drug Development, 192 South Street, Suite 550,Boston, Massachusetts 02111, USA e-mail: janice.reichert@tufts.edu

portfolio of 73 marketed products1,2. 65 D&E 47 The analysis of the rDNA protein sector 42 AIID presented here discloses 60 key trends that have 24 guided the current marCV/hemotosis 27 ket formation and that will affect future mar33 Anti-infective 17 ket growth. Our analysis included a thorough 13 Antineoplastic evaluation of thesector’s 15 historical approval suc35 All products cess rates, followed by 26 market analysis and rev40 60 20 0 10 30 50 70 enue forecasts for the U.S. approval success rate (%) current key companies, 1990-1997 (n=120*) 1980-1989 (n=91*) therapeutic foci and leading protein classes. Only novel rDNA therapeutic Figure 1 US approval success rates for rDNA therapeutics in two periods. products wereevaluated; *Total number of products that entered clinical study during period. AIID, variants of innovative arthritis, inflammation and immune disorders; CV, cardiovascular; D&E, diabetes and endocrinology. Source: Tufts Center for the Study of Drug recombinant products Development. (e.g., reformulations) and rDNA products developed as therapeutic vaccines were excluded. Therapeutic monoclo- Thatwas then—the 1980s nal antibodies were also excluded; success rates The development of methods to manipulate and market values for these products have been DNA opened the door to virtually unlimpublished3–5. ited possibilities for recombinant protein We find that, as the biotechnology industry production. In theory, the only limit was developed and early rDNA products fulfilled imagination. Inpractice, the prevailing biotheir promise in the market, the resulting logical knowledge and projected market size growth of the rDNA market guided the forma- for rDNA therapeutics dictated the focus of tion and composition of the current pipeline early efforts. The proteins studied in the clinic of products. Analysis of key trends relating to in the 1980s were those that succumbed to approved and...