ABSTRACT
Biopharmaceuticals are proteins and nucleic acid-derived molecules that are used for therapeutic purposes or in vivo diagnostics, and are produced by means other than direct extraction from a native (non-engineered) biological source (Walsh, 2002). When considered together with vaccines and biomolecules extracted from natural sources, they are designated ‘‘biologicals’’ by the pharmaceutical industry. A survey carried out by the Pharmaceutical Research and Manufacturers
of America (PhRMA) verified that in 2004 over 100 biopharmaceuticals had already gained approval from the US Food and Drug Administration (FDA) (PhRMA, 2004). This survey found that 324 new biotechnology medicines, targeting nearly 150 diseases, were undergoing human clinical trials or under review by the FDA. Among the products under development, almost half (154) were intended for cancer treatment, and another significant portion for infectious diseases, AIDS/HIV and related conditions, and autoimmune and neurological disorders. Despite the long development time for biopharmaceuticals (Figure 16.1) and the fact that most molecules under clinical trials do not gain final approval from the regulatory agencies, a considerable increase in the number of commercialized biopharmaceuticals is expected in the next few years. It is estimated that to date more than 250 million people worldwide
have benefited from the biologicals already approved for the treatment or prophylaxis of cardiac diseases, multiple sclerosis, several types of cancer, hepatitis, arthritis, and diabetes, among others. Since biopharmaceuticals focus their action on the molecular basis of diseases, they are providing physicians and patients with new powerful tools for the treatment of diseases, changing fundamentally the way diseases are treated (PhRMA, 2004).
