UNIT-1 AM
1.1 - Pharmaceutical Products
Pharmaceutical Products also referred to as a medicine or medicinal product, It can be
loosely defined as any chemical substance or product comprising such intended
for use in the medical diagnosis, cure, treatment, or prevention of disease. Pharmaceutical
substances form the backbone of modern medicinal therapy.
Characteristics
Most traditional pharmaceuticals are low molecular weight organic chemicals. Although
some (e.g. aspirin) were originally isolated from
biological sources, most are now manufactured by direct chemical synthesis.
1.Efficacy – The drug must effectively treat the intended condition.
2.Safety – Should have minimal adverse effects.
3.Quality – Must be manufactured under strict guidelines (GMP).
4.Stability – Should maintain potency over time.
Manufacturing company
• Two types of manufacturing company thus comprise the ‘traditional’ pharmaceutical
sector:
• The chemical synthesis plants, which manufacture the raw chemical ingredients in bulk
quantities, and the finished product
pharmaceutical facilities, which purchase these raw bulk ingredients, formulate them into
final pharmaceutical products, and supply these products to the end user.
Drug from Biological sources
In addition to chemical-based drugs, a range of pharmaceutical substances are produced
by from biological sources. And Nowadays using genetic engineering /GMO such products
are also referred as a biotechnological products
• Hormones • Blood products
• Peptides • Proteins
• Enzymes • Nucleic acid
Examples:
1. Insulin – Obtained from bacteria using recombinant DNA technology.
2. Penicillin – Derived from Penicillium mold.
Biotech products
-Biotech products are derived from living organisms such as bacteria, yeast, or mammalian
cells
-Biotech products are typically large, complex molecules like proteins or monoclonal
antibodies, making them difficult to characterize fully.
-Biotech products require advanced techniques like fermentation, genetic engineering, and
cell culture, which involve living cells.
Chemical products
-chemical products are synthesized through chemical reactions using raw materials like
petroleum derivatives or inorganic compounds.
-chemical products are small, well-defined molecules with a simpler structure.
-are manufactured through precise chemical synthesis, often involving multiple controlled
reactions.
Examples: Biotech products include insulin and monoclonal antibodies, while chemical
products include aspirin and paracetamol.
Semisynthetic
These are chemically modified versions of natural drugs to improve efficacy, reduce
toxicity, or enhance stability.
For example, certain semi-synthetic antibiotics (Amoxycillin,ampicillin) are produced by
chemical
modification of natural antibiotics produced by fermentation technology (penicillin).
Well-Characterised Biotech products
1. Synthetic peptides (>20 aminoacid)
2. MAbs and derivatives
3. rDNA derived products
4. DNA and plasmid based product
1.2 biopharmaceutical and pharmaceutical company
The term ‘biopharmaceutical’ was first used in the 1980s and came to describe a class of
therapeutic proteins produced by modern biotechnological techniques,
specifically via genetic engineering or, in the case of monoclonal antibodies, by hybridoma
technology
• Although the majority of biopharmaceuticals or
biotechnology products now approved or in development
are proteins produced via genetic engineering, these terms now also encompass nucleic-
acid-based, i.e.
deoxyribonucleic acid (DNA) or ribonucleic acid (RNA)-based products, and whole-cell-
based products.
1.3 History of pharmaceutical industry
Pharmaceutical industry is 60 to 70 year old. Grow rapidly and estimated value of US$100
billion by the mid 1980s. There are well in excess of 10 000 pharmaceutical companies in
existence, although only about 100
of these can claim to be of true international significance. Same in India where thousand
players are there and few are internationally important.
These companies manufacture in excess of 5000 individual pharmaceutical substances
used routinely in medicine.
20th century development
• The first stages of development of the modern pharmaceutical industry can be traced back
to the turn of the twentieth century. At that time (apart from folk cures), the
medical community had at their disposal only four drugs that were effective in treating
specific disease.
4 drugs
•Digitalis (extracted from foxglove) was known to stimulate heart muscle and, hence, was
used to treat various heart conditions.
• Quinine, obtained from the barks/roots of a plant (Cinchona genus), was used to treat
malaria
• Pecacuanha (active ingredient is a mixture of alkaloids), used for treating dysentery, was
obtained from the bark/roots of the plant genus Cephaelis.
• Mercury, for the treatment of syphilis.
Landmark discovery
• Despite these early advances, it was not until the 1930s that the pharmaceutical industry
began to
develop in serious.
• The initial landmark discovery of this era was probably the discovery, and chemical
synthesis, of the sulfa drugs by Domark.
• These are a group of related molecules derived from the red dye prontosil rubrum.
Early drugs
These drugs proved effective in
the treatment of a wide variety
of bacterial infections Although
of bacterial infections Although
it was first used therapeutically
in the early 1920s, Large-scale industrial production of insulin also
commenced in the 1930s.
The medical success of these drugs gave new emphasis to the pharmaceutical industry,
which was boosted further by the commencement of industrial-scale
penicillin manufacture in the early 1940s.
Pharmaceutical companies and new
drugs
• Around this time, many of the current leading pharmaceutical companies (or their
forerunners) were founded.
• Examples include Ciba Geigy, Eli Lilly, Wellcome, Glaxo and Roche.
• Over the next two to three decades, these companies developed drugs such as
tetracyclines, corticosteroids, oral
contraceptives, antidepressants and many more.
Extensive prescribed drug and tragedy
• Diazepam (Valium) (used to treat anxiety, panic attacks), discovered in 1960, was marketed
from 1963 and rapidly became the most prescribed drug in history, prior to
controversy over dependency and
habituation.
Key discoveries leads to develop the
pharma industries
• Most of today's major pharmaceutical companies were founded in the late 19th and early
20th centuries. Key discoveries of
the 1920s and 1930s, such as insulin and penicillin, became mass-manufactured and
distributed. Switzerland, Germany and Italy
had particularly strong industries, with the United Kingdom, the United States, Belgium and
the Netherlands following suit.
1.5 Biopharmaceutical and current status and future
prospects
Approximately one in every four new drugs now coming on the market is a
biopharmaceutical. By mid 2006, some 160 biopharmaceutical products had gained
marketing approval in the USA and/or EU.
Collectively, these represent a global biopharmaceutical market in the region of US$35
billion, and the market value is estimated to surpass US$50 billion by 2010. Strategic
Analysis & Forecast of US Biopharmaceuticals suggests the U.S. biopharmaceuticals market
is expected to increase to $144 billion by 2016.
Protein based products
The products include a range of hormones, blood factors and
thrombolytic agents, as well as vaccines and monoclonal antibodies. All but two are protein-
based therapeutic agents. The exceptions are two nucleic-acid-based products: ‘Vitravene’,
an
antisense oligonucleotide, and ‘Macugen’, an aptamer.
Nucleic acid based product
Many additional nucleic-acid-based products for use in gene therapy or antisense
technology are in clinical trials, although the range of technical difficulties that still beset
this class of therapeutics will ensure that protein-based products will overwhelmingly
predominate for the
foreseeable future.
Host cells for engineered protein
Thus far, the vast majority of approved recombinant
proteins have been produced in the
– E. coli,
– Yeast S. cerevisiae, Pichia etc
– In animal cell lines (Chinese hamster ovary (CHO) cells or baby hamster kidney (BHK) cell.
Produced by engineered / transgenic organisms
Although most protein-based products likely to gain marketing approval over the next 2–3
years
will be produced in engineered E. coli, S. cerevisiae or animal cell lines, some products now
in clinical
trials are being produced in the milk of transgenic animals. Additionally, plant-based
transgenic expression
systems may potentially come to the fore, particularly for the production of oral vaccine.
systems may potentially come to the fore, particularly for the production of oral vaccine.
Companies
Companies already producing generic biopharmaceuticals
include
– Biopartners (Switzerland),
– Genemedix (UK),
– Sicor and Ivax (USA),
– Congene and Microbix (Canada)
– BioGenerix (Germany)
Aptamers
Aptamers (from the Latin aptus - fit, and Greek meros - part
• Aptamers represent an additional emerging class of nucleic-acid-based therapeutic.
• These are short DNA- or RNA-based sequences that adopt a specific three-dimensional
structure, enabling them to bind (and thereby inhibit) specific target
molecules.
• One such product (Macugen) has been approved to date.
