Biotechnology Products & Regulatory requirements

Medical Writer Group NLNaarden, 8 Dec 2005

H.F. Schuring

Definition of Biotechnology

• Bio – deals with living organisms

• Technology – involves the practical application of knowledge

Therefore, Biotechnology is the practical application of knowledge related to living

organisms

Biotech vs Biologicals

• Biologicals are products which quality, purity, specificity, and potency cannot be expressed completely by chemical and physical terms (UK approach)

• Origin, manufacture and structure are not mentioned, therefore includes– immunological products (vaccines, toxins, serum,

and allergens -89/342/EC)– products from human blood or plasma -

89/381/EC

Wide Range of Products

• New molecular entities:– Recombinant DNA products

• CHO products• Proteins from milk of transgenic animals

– Gene therapy products

– Cell therapy products

– Monoclonal antibodies

Evolution of Biotechnology

10,000 BC: Alcohol fermentation, bread, cheese & yogurt

5,000 BC: Beer is brewed in Egypt

Evolution of Biotechnology (Overview)

• Whole Organism (macroscopic level)• Cells and other Microorganisms• Subcellular

– Components of the cell (organelles)– What did these components do?

• Nucleus and its Genetic Material– How did this material control the cell?– How was this information passed on

as the cell divided?

1953

Watson & Crick resolve the double helix structure of DNA

Evolution of Biotechnology 1800 - today

Key Developments in Biotechnology

• Cell Culture (bacteria, yeast, CHO)

• Monoclonal Antibodies

• Molecular Biology– Genetic Engineering– Recombinant DNA Technology– Transgenic Animals– Cloning

• Stem Cells (Great Potential)

Commercial Production

Level Control

Medium Product BaseBioreactor with Packed Bed

Addition

HarvestAddition

Quality

Purity - Impurity

Biological properties

Immunological properties

Strength

Potency

Safety Efficacy

Implication of the manufacturing process on a r-DNA biotech product

• The process defines the product

and

• The process determines the quality and reproducibility

Note: case by case assessments

Regulatory Requirements

• Quality, Safety and Efficacy– Less guidance available compared versus NCEs

• Quality specifics:– Characterization– Viral safety– Cell banking

• Preclinical specifics:– ICH S6 versus ICH M3– Immunogenicity

• Clinical specifics:– dependent on product– Immunogenicity