Biopharmaceuticals PDF

Biopharmaceuticals Dr. Shatavari Kulshrestha Assistant Professor Introduction The pharmaceutical and biopharmaceutical industry sector companies engaged in researching, developing, manufacturing, and distributing drugs  Drugs are products intended for use in the diagnosis, cure, mitigation...

Biopharmaceuticals Dr. Shatavari Kulshrestha Assistant Professor Introduction The pharmaceutical and biopharmaceutical industry sector companies engaged in researching, developing, manufacturing, and distributing drugs  Drugs are products intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease and products (other than food) intended to affect the structure or any function of the body The definition also includes components of drugs, such as active pharmaceutical ingredients and excipients  Excipient Excipient is a substance formulated alongside the active ingredient of a medication serve various purposes, including long-term stabilization bulking up solid formulations containing potent active ingredients in small amounts enhancing the therapeutic properties of the active ingredient in the final dosage form Excipient They can facilitate drug absorption, reduce viscosity, or enhance solubility Also aid in the manufacturing process by improving the handling of active substances, facilitating powder flowability, or preventing denaturation and aggregation during the expected shelf life The selection of excipients depends on factors such as the route of administration, dosage form, and active ingredient Pharmaceutical substances Low molecular mass organic chemicals formed by direct chemical synthesis (less than 900 Daltons) Chemical synthesis plants (Raw chemical ingredients, bulk quantities) Finished product pharmaceutical facilities (Formulation of raw bulk material into final product and supply to end user) Reference: Gary Wash (2011) Biopharmaceuticals: Biochemistry and biotechnology , second edition Biopharmaceuticals The term ‘biopharmaceutical’ was first used in the 1980s a class of therapeutic protein produced by modern biotechnological techniques, specifically via genetic engineering or (in the case of monoclonal antibodies) by hybridoma technology The recombinant human insulin (trade name “Humulin”) was the first biopharmaceutical approved for human therapeutic uses and marketing in 1982 Biopharmaceutical definition The term ‘biopharmaceutical’ refers to protein or nucleic acid based pharmaceutical substances used for therapeutic or in vivo diagnostic purposes, which are produced by means other than direct extraction from natural (non- engineered) biological sources Reference: Gary Walsh (2011) Biopharmaceuticals: Biochemistry and biotechnology , second edition Biologic medicinal products derived from blood, as well as vaccines, toxins and allergen products Biotechnology medicine Products of pharmaceutical biotechnology’ or ‘biotechnology medicines’ Any pharmaceutical product used for therapeutic or in vivo diagnostic purposes, which is produced in full or in part by biotechnological means Biosimilar A biosimilar product is a biologic product that is approved based on demonstrating that it is highly similar to an FDA‐approved biologic product, known as a reference product, and has no clinically meaningful differences in terms of safety and effectiveness from the reference product Only minor differences in clinically inactive components are allowable in biosimilar products  Biotechnology Biotechnology medicine any pharmaceutical product used for a therapeutic refers to the use of biological systems (e.g. cells or or in vivo diagnostic purpose, which is produced in tissues) or biological molecules (e.g. enzymes or full or in part by either traditional or modern antibodies) for or in the manufacture of commercial biotechnological means for example, antibiotics extracted from fungi, therapeutic products. proteins extracted from native source material (e.g. insulin The term is equally applicable to long-established from pig pancreas) and products produced by biological processes, such as brewing, and more genetic engineering (e.g. recombinant insulin) modern processes, such as genetic engineering Biologics refers to medicinal products derived from blood, as well as Biopharmaceutical vaccines, toxins and A protein or nucleic acid based pharmaceutical allergen products. substance used for therapeutic or in vivo diagnostic Thus, some traditional biotechnology-derived purposes, which is produced by means other than pharmaceutical products (e.g. direct extraction from a native (non-engineered) hormones, antibiotics and plant metabolites) fall biological source outside the strict definition.  Biotechnology medicine Biologics Biopharmaceut icals Reference: Gary Walsh (2011) Biopharmaceuticals: Biochemistry and biotechnology , second edition  History of the pharmaceutical industry 20th 1800s 1920s 1940s 1950s century to 1900s Four drugs Therapeutically Discovery and Industrial-scale Proteins important chemical penicillin produced Digitalis (foxglove) artificial dyes synthesis of manufacture naturally in for heart condition sulfa drugs body which ahs Formation of derived from Ciba Geigy, Eli therapeutic Quinine (Cinchona pharmaceutical red dye, Lilly, Wellcome, applications sp.) for malaria companies like Prontosil Glaxo and Bayer and rubrum for Roche Interferon, Pecacuanha Hoechst bacterial interleukins , (Cephalis) for infection Drugs such as erythropoietin, dysentery Bayer treatment tetracyclines, neurotrophic succeeded in corticosteroids, factors Mercury Calomel synthesizing Large scale oral (Mercurous aspirin in 1895 production of contraceptives, Production in chloride) for syphilis insulin in 1930s antidepressants small quantities Recombinant DNA technology has four fold positive impact on production of pharmaceutically important proteins Overcome the problem of source availability Recombinant production allows the manufacture of any protein in whatever quantity it is required Many proteins of therapeutic potential are produced naturally in the body in minute quantities Interferons, interleukins and colony stimulating factors Not sufficient to meet clinical demands Overcomes problems of product safety Direct extraction from native source may lead to transmission of diseases The transmission of blood-borne pathogens such as hepatitis B, C and HIV via infected blood products Transmission of Creutzfeldt–Jakob disease to persons receiving human growth hormone preparations derived from human pituitaries Alternative to direct extraction from inappropriate/dangerous source material Fertility hormone FSH is obtained from the urine of post- menopausal women and Hormone hCG, is extracted from the urine of pregnant women Ancrod, for example, is a protein displaying anti-coagulant activity; however, it is produced naturally by the Malaysian pit viper Retrieval by milking snake venom is possible But RDT production preferable Facilitates the generation of engineered therapeutic proteins displaying some clinical advantage over the native protein product Site-directed mutagenesis Insertion, deletion or alteration of a single amino acid residue The alteration or deletion of an entire domain, or the generation of a novel hybrid protein Reference: Gary Wash (2011) Biopharmaceuticals: Biochemistry and biotechnology , second edition Many protein-based products extracted directly from native source material remain on the market Economic reason, e.g. if the protein is produced in very large quantities by the native source and is easy to extract/purify e.g. human serum albumin Some blood factor preparations purified from donor blood actually contain several different blood factors and hence can be used to treat several haemophilia patient types Recombinant blood factor preparations contain a single blood factor and hence can be used to treat only one haemophilia type Era of Biopharmaceuticals Late 1970s and early 1980s The advent of genetic engineering and monoclonal antibody technology underpinned the establishment hundreds of start- up biopharmaceutical companies The merger of biotech capability with pharmaceutical experience helped accelerate development of the biopharmaceutical sector (Genentech produced recombinant insulin later marketed by Eli Lilly under trade name Humulin) Amgen, Biogen and Genentech represent three pioneering biopharmaceutical companies that remain in business