Large molecule drug

Table 1.2 shows the top 10 drugs in 2006 with the exception of Enbrel (a biopharmaceutical, large molecule drug—see Chapter 4), all the others are... 

Unlike the small molecule drugs (pharmaceuticals) described in Chapter 3, large molecule drugs (biopharmaceuticals) are mainly protein based. Another distinction is that these protein-based drugs are, in the main, similar to natural biological compounds found in the human body or they are fragments that mimic the active part of natural compounds. 

Demonstrate by citing examples the current treatment of breast cancer using small and large molecule drugs. 

Exhibit 5.13 Selected Large Molecule Drug Formulations... 

Examples of PD, PK, toxicology, and formulation for two selected drugs, Zyprexa (a small molecule drug) and Aranesp (a large molecule drug) are described next. 

Refer to Sections 7.2.1 and 7.2.2 about the CDER and CBER. It should be understood that small and large molecule drugs are legislated differently the former under the Food, Drug and Cosmetic Act (FDCA) and the latter under the Public Health Service Act (PHSA). 

Refer to Section 8.2.3. The NDA is for small molecule drugs and the BLA is for large molecule drugs. They are legislated under different acts. 

Refer to Section 8.3.2. Note the requirement for the Qualified Person and Expert Reports in Europe. There are no separate applications in the Centralized Procedure for small and large molecule drugs. 

There are distinct differences in the manufacture of small and large molecule drugs the former is mainly based on organic chemical synthesis, while the latter relies on biological systems of recombinant DNA (rDNA) technology. In this chapter, we describe the manufacturing processes for these drugs under GMP environments. 

The need to transport temperature-sensitive raw materials and products, such as cell line, medium, large molecule drugs, and vaccines, means that some form of control during transportation is needed. For example, a working cell bank for the production of proteins may be transported in liquid nitrogen (-196 °C) and that of protein and vaccines in dry ice (-78 °C) in order to protect the integrity of the materials. Data loggers are used to record the temperature... 

Small molecule drugs are produced using organic synthesis processes whereas large molecule drugs are derived mainly from living cells, such as microbial and mammalian cells. 

Refer to Sections 10.1,10.4, and 10.5 the organic synthesis route is used for small molecule drugs and living cells for large molecule drugs. 

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