Market approval

Thrombopoietic factors (no recombinant TPO product in clinical use at this time IL-11 [recombinant product oprelvekin] has marketing approval) stimulate the production of megakaryocyte precursors, megakaryocytes, and platelets [8]. Interleukin-11 has many effects on multiple tissues, and can interact with IL-3, TPO, and SCF. AMG 531, a recombinant peptibody in that binds to the thrombopoetin receptor Mpl and stimulates the production of platelets, is in phase 1 and 2 studies and has been shown to safely increase platelet counts in patients with immune thrombocytopenic purpura [9]. 

Maximum patent extension 15 from first marketing approval 14 from marketing approval... 

The 510(k) pre-market notification process is not as onerous as the pre-market approval procedure, as clearance to market a device is not based on actual assessment of the safety and effectiveness of the particular device in question. Instead, devices can be cleared on the basis that they are substantially equivalent to existing devices that have been recognised as safe and effective, or that they conform to specific device standards promulgated or recognised by the FDA. There are four procedural variations to the 510(k) notification process. 

This is not a common approach, as new innovative devices will tend to be viewed as Class III devices requiring pre-market approval. However, if the manufacturer can show that the level of risk does not warrant placing it in the higher risk category, he/she may petition the FDA to have it reviewed through the de novo 510(k) procedure. 

The pre-market approval process is a considerably more complex procedure, as the FDA can only grant marketing approval on the basis of an assessment of the actual safety and effectiveness of the device in question. Thus, it is similar to a drug... 

Investigational Device Exemptions - 21 CER Part 812 Pre market approval - 21 CFR Part 814... 

Part 71 Color additive petitions — These regulations describe the pre-market approval process for new color additives or new uses for listed color additives. ... 

Before a marketing approval document, such as an NDA or ANDA, is finally approved by FDA, an inspection by investigators of the local district office will be arranged [3]. This inspection can provide an additional level of assurance that a generic product will indeed meet all required quality standards. 

Table 1.3 Selected engineered biopharmaceutical types/products that have now gained marketing approval. These and additional such products will be discussed in detail in subseguent chapters...

The Committee for Medicinal Products for Human use (CHMP). This committee is composed of 35 technical experts drawn from the various EU member countries. It is primarily responsible for formulating the EMEA s opinion on any medicinal product being considered for marketing approval under the centralized procedure. 

Under the centralized procedure, applications are accepted with regard to (a) products of biotechnology (b) NCEs (drugs in which the active ingredient is new). Biotech products are grouped as list A and NCEs as list B . Marketing approval application for biotech products must be considered under the centralized procedure, whereas NCEs can be considered under centralized or decentralized mechanisms. 

The expression of recombinant proteins in cells in which they do not naturally occur is termed heterologous protein production (Chapter 3). The first biopharmaceutical produced by genetic engineering to gain marketing approval (in 1982) was recombinant human insulin (tradename Humulin ), produced in E. coli. An example of a more recently approved biopharmaceutical that is produced in E. coli is that of Kepivance, a recombinant keratinocyte growth factor used to treat oral mucositis (Chapter 10). Many additional examples are provided in subsequent chapters. 

Three additional recombinant hormones have recently gained marketing approval thyroid-stimulating hormone, parathyroid hormone and calcitonin. 

A number of such recombinant (subunit) vaccines have now been approved for general medical use (Table 13.9). The first such product was that of hepatitis B surface antigen (rHBsAg), which gained marketing approval from the FDA in 1986. Two billion people are infected with hepatitis B worldwide, 350 million individuals suffer from life-long chronic infection, and more... 

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