BiopharmaceuticalsTransforming proteins and genes into drugs                           By-                           Kulde...
Flow of presentation      I. Biopharmaceutical        1.Definition        2 Structure        3.Type        4. Manufacturin...
Terminology used     Biologics     Biopharmaceuticals     Biosimilars     Follow-on Biologics     Biogenerics3   Note...
Cornerstones in biotechnology history which have influenced the    production of Biopharmceuticals    1953: Discovery of D...
Traditional Vs Biopharmceuticals          Traditional                   Biopharmceuticals        Multiple effect         ...
Definitions of BiologicsBiopharmaceuticals       A biologic can be any therapeutic serum, toxin, antitoxin, vaccine, virus...
Complexity of Biopharmceuticals                                          Structure     Epoetin              Size          ...
Types of Biologics     Peptides     Non-glycosylated proteins     Glycosylated proteins     Monoclonal antibodies8
Manufacturing Process                            Cell Bank                            UPSTREAM                      •Cell ...
Biotech Process                A typical fermentation based biotech                             process flow10
FDA Approvals (2005-2009) Synthetic V/s Biopharmceuticals                             25                                  ...
EMEA Approvals (2005-2009) Synthetic V/s Biopharmceuticals                             25                                 ...
Benefits of Biopharmaceuticals      Highly effective and potent action      Fewer side effects      Potential to actual...
Top 10 Biopharmceuticals in 2008     #Rank   Biopharmceuticals    Brands          Company                  Sales(08)(US$ m...
Top 10 companies in Biopharmceuticals                                 Sales/Revenues     R & D spending     # Rank       C...
Cost of Biopharmceuticals                       Drug                                      Indication                   Cos...
Biosimilar
Definitions and Terminologies      A biosimilar medicine is a medicine which is similar to a biological medicine that has ...
Biosimilars -       Scientific basis for abbreviated pathway                     Demonstrate Quality, Safety, Efficacy    ...
Regulation of Biosimilars
No Harmonized Worldwide Regulatory Framework for      Biosimilars      Small molecule generics model is inappropriate    ...
Biosimilars – Regulatory Perspective         Well Defined Framework                               Under process of Develop...
Biosimilars In the Market Today      Europe - Sandoz –Omnitrope (hGH), Binocrit (“EPO” or erythropoietin);       Biopartn...
Strategic Options to Tap Biosimilar Market      Generics firms enter successfully into the biosimilars                    ...
Biosimilars successful if all hurdles passed25
The other side of the Biopharmceuticals…….     Safety issue …………??????????      Biologic drugs are orders of magnitude mo...
Key Success Factors for Biosimilars       1.   Consistent long term strategy       2.   Healthy financial structure       ...
Regulation of Biologics
Challenges in front of Biopharmceuticals        From Industry perspective      Long and costly clinical trials      Effi...
Continue…          From Govt. perspective      No guidelines (Since Biopharmceuticals are in Nascent stage)       From Pa...
Future of Biopharmceuticals      More diseases will come under the treatment      Effective manufacturing31
Conclusion      Biopharmceuticals or PERISH!!!!!!!      Traditional drug1st generation Biopharmceuticals (small       m...
33
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Biopharmaceuticals (Transforming proteins and genes into drugs)

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A outline of Biopharmaceutical market of the World

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  • Source: http://www.egagenerics.com/doc/biosimilars_helsinki_2008-10-31.pdf http://www.pmda.go.jp/english/past/2009bio_sympo/file/I-2_Nomura(JPMA).pdf http://www.diahome.org/product/16920/Kim_18430_852.pdf http://www.slideshare.net/m_a_staples/MStaplesBioGenerics-HandoutMAR08
  • http://www.fda.gov/BiopharmceuticalsBloodVaccines/DevelopmentApprovalProcess/BiologicalApprovalsbyYear/default.htm http://www.fda.gov/BiopharmceuticalsBloodVaccines/DevelopmentApprovalProcess/BiologicalApprovalsbyYear/default.htm
  • http://www.ema.europa.eu/htms/human/epar/a.htm Thomson Partners; IDDB3 IMS Health
  • Source- http://www.contractpharma.com/articles/2009/07/2009-top-10-biopharmaceutical-companies-report
  • http://www.managedcaremag.com/supplements/0803_bfac/BFAC_monograph_MC.pdf http://www.law.duke.edu/journals/dltr/articles/2008dltr0009.html
  • Source: http://www.egagenerics.com/doc/biosimilars_helsinki_2008-10-31.pdf http://www.pmda.go.jp/english/past/2009bio_sympo/file/I-2_Nomura(JPMA).pdf http://www.diahome.org/product/16920/Kim_18430_852.pdf http://www.slideshare.net/m_a_staples/MStaplesBioGenerics-HandoutMAR08
  • http://www.scripnews.com/home/Japans-biosimilar-guidelines-edge-forward-115782 http://www.genengnews.com/articles/chitem.aspx?aid=2099&chid=4 Evaluation of Medicines for Human Use http://www.prlog.org/10273398-negotiating-the-emerging-biosimilars-landscape-key-developments-in-the-regulatory-environment.html 5. http://www.pjbpubs.com/uploads/RycPYX13U757v7h2kpec.pdf ( page 17-20) 6. http://www.scribd.com/doc/2068017/Biosimilars-Overview-18012007-v2
  • http://www.biosimilarstoday.com/2008/King.pdf
  • http://www.accenture.com/NR/rdonlyres/B5765AC2-4B74-4512-9034-BB4420A6BC5D/0/Accenture_HLS_PMP_Biosimilars.pdf
  • Biopharmaceuticals (Transforming proteins and genes into drugs)

    1. 1. BiopharmaceuticalsTransforming proteins and genes into drugs By- Kuldeep Dabasby: Prepared XIIIth Batch
    2. 2. Flow of presentation I. Biopharmaceutical 1.Definition 2 Structure 3.Type 4. Manufacturing 5. Top 10 Biopharmaceuticals(2008) 6. Top 10 Biopharmaceutical companies II. Biosimilars 1.Definition 2.Regulation 3.Strategies III. Challenges for Biologics IV. Future V. Conclusion2
    3. 3. Terminology used  Biologics  Biopharmaceuticals  Biosimilars  Follow-on Biologics  Biogenerics3 Note- Following terms are used in different organization in different places
    4. 4. Cornerstones in biotechnology history which have influenced the production of Biopharmceuticals 1953: Discovery of DNA structure 1973: Discovery of DNA restriction enzymes 1977: Genentech, first biotech-enterprise founded 1982: First biopharmaceutical approved by FDA: recombinant human insulin 1986: First recombinant vaccine (HepB) is approved for human use, first recombinant anti-cancer drug (Interferon) is produced 2003: Human genome sequenced4
    5. 5. Traditional Vs Biopharmceuticals Traditional Biopharmceuticals Multiple effect Specific effect Short Acting Long acting Non-Immunogenic Immunogenic Species independent Species dependent Small molecules Large molecules Stable Heat sensitive Oral administration Parenteral General practice Hospital5
    6. 6. Definitions of BiologicsBiopharmaceuticals A biologic can be any therapeutic serum, toxin, antitoxin, vaccine, virus, blood, blood component or derivative, allergenic product, or analogous product, or derivatives applicable to the prevention, treatment, or cure of injuries or disease of human Source – FDA “A biological substance is a substance that is produced by or extracted from a biological source and for which a combination of physico-chemical-biological testing and the production process and its control is needed for its characterisation and the determination of its quality Source – EMEA “a substance which cannot be completely characterized by physicochemical means alone and which therefore requires the use of some form of bioassay”. Source – WHO6 Note: Sources mentioned in the notes section of slide
    7. 7. Complexity of Biopharmceuticals Structure Epoetin Size Aspirin M Stability od ifi ca tio n7
    8. 8. Types of Biologics  Peptides  Non-glycosylated proteins  Glycosylated proteins  Monoclonal antibodies8
    9. 9. Manufacturing Process Cell Bank UPSTREAM •Cell expansion •Fermentation Raw In •Clarification materials process DOWNSTREAM •Centrifugation •Chromatography •Ultra filtration Drug substances Drug release substances9
    10. 10. Biotech Process A typical fermentation based biotech process flow10
    11. 11. FDA Approvals (2005-2009) Synthetic V/s Biopharmceuticals 25 21 20 19 18 18 16 Number of Approvals 15 10 6 5 4 3 2 2 0 2005 2006 2007 2008 2009 New molecular entities New biologic entities11
    12. 12. EMEA Approvals (2005-2009) Synthetic V/s Biopharmceuticals 25 23 20 20 19 16 Number of Approvals 15 13 10 10 8 6 5 5 4 0 2005 2006 2007 2008 2009 New molecular entities New biologic entities More number of Biopharmceuticals were approved by EMEA as compared to FDA in 200912
    13. 13. Benefits of Biopharmaceuticals  Highly effective and potent action  Fewer side effects  Potential to actually cure diseases rather than merely treat the symptoms  Longer half life13
    14. 14. Top 10 Biopharmceuticals in 2008 #Rank Biopharmceuticals Brands Company Sales(08)(US$ million) Therapeutic area 1 Etanercept Enbrel Amgen, Wyeth $6,580 Rheumatoid Arthritis J&J, Schering Plough, 2 Infliximab Remicade $5,934 Rheumatoid Arthritis Mitsubishi 3 Bevacizumab Avastin Roche $5,777 Colorectal cancer. 4 Rituximab Rituxan Roche $5,653 Head and Neck Cancer 5 Adalimumab Humira Abbott $5,488 Rheumatoid Arthritis 6 Epoetin alfa Epogen Amgen $5,033 Renal anemia 7 Trastuzumab Herceptin Roche $4,890 Breast cancer 8 Insulin Lantus Sanofi Aventis $4,180 Diabetes 9 Pegfilgrastim Neulasta Amgen $3,355 Neutropenia 10 Darbepoetin Aranesp Amgen $2,871 Anemia14 Source- La Merie Business intelligence (R&D Pipeline news 10 March 2010)
    15. 15. Top 10 companies in Biopharmceuticals Sales/Revenues R & D spending # Rank Company Therapeutic area In US$ Million) (In US$ Million) 1. Amgen 14,687 $2,900 Oncology, kidney disease, rheumatoid arthritis Oncology, Immunology, Tissue Repair, Neuroscience, Ophthalmology 2. Genentech 10,531 2,800 Diabetes care , Haemostatic management , Growth hormone 3. Novo Nordisk 8,989 1,550 therapy , Hormone replacement therapy Neurodegenerative Diseases, Oncology, Fertility, Endocrinology, 4. Merck Serono 7,338 1,580 Autoimmune and Inflammatory, Cardio metabolic care Baxter biopharma 5. 5,308 868 Hemophilia, Biotherapeutics, Regenerative Medicine, Vaccines solutions 6. Biogen Idec 3,968 1,072 Neurology, Oncology, Immunology, Hemophilia, Cardiopulmonary Genetics Diseases, Cardio metabolic and Renal, Oncology, 7. Genzyme 3,751 750 Orthopaedics/Biosurgical Specialties, Transplant, Genetics/Diagnostics Dermatology, Urology, Cardiovascular, Antibiotics, Anaphylaxis, 8. CSL ltd 2,961 202 Central nervous system, Analgesia, Emergency Care, Obstetrics and Gynecology Neurosciences, Medical Dermatology and Urology, Eye Care, 9. Allergan 1,311 798 Medical Aesthetics, Obesity Intervention 10. Alexion Pharma 259 63 Hematology15 Source-Contract Pharma Articles » 2009 » July/August 2009
    16. 16. Cost of Biopharmceuticals Drug Indication Cost Duration Cerezyme Life-threatening enzyme deficiency $200,000 to $500,000 12 Month Trastuzumab (Herceptin) Breast Cancer $36,000 6 Month Rituximab (Rituxan) Non-hodgkin’s lymphoma $32,500 2 Month Bevacizumab (Avastin)/ Cetuximab Metastatic Colorectal Cancer $28,500 2 Month (Erbitux) Infliximab (Remicade), Rheumatoid arthritis $18,000 - Infliximab Crohn’s disease $16,500 - Cost for indication listed are for 2005 Source- MONROE 200616
    17. 17. Biosimilar
    18. 18. Definitions and Terminologies A biosimilar medicine is a medicine which is similar to a biological medicine that has already been authorised (the ‘biological reference medicine’)1 Source – EMEA “A follow-on biologic (FOB) is “a protein product which is intended to be a similar version or duplicate of an already approved or licensed protein product”.4 Source – FDA A new biological medicinal product claimed to be “similar” with regard to quality, safety and efficacy to an already approved reference medicinal product3 Source – WHO “A drug to be developed by a different marketing approval holder as a drug that is bio-equivalent/quality-equivalent to biotechnology-derived drug already approved domestically” 2 Source – PMDA ,Japan Commonly used terminologies/synonyms • Biosimilars • Biogenerics • Follow on Biopharmceuticals • Follow on Protein18 Note: Sources mentioned in the notes section of slide
    19. 19. Biosimilars - Scientific basis for abbreviated pathway Demonstrate Quality, Safety, Efficacy Surveillance Regulatory Extensive Approval New Biologic Clinical Characterization Pre-Clinical Clinical Extensive Regulatory Surveillance Characterization Approval Clinical Biosimilar Extensive Pre-Clinical Comparison to Pre-Clinical Reference Allows for abbreviated19 pre-clinical & clinical
    20. 20. Regulation of Biosimilars
    21. 21. No Harmonized Worldwide Regulatory Framework for Biosimilars  Small molecule generics model is inappropriate  In many regions limited or no regulatory processes exist  Lack of minimum regulatory standards presents a risk for patients because of the potential issues relating to the quality, efficacy and safety of biosimilars developed and approved without defined requirements21
    22. 22. Biosimilars – Regulatory Perspective Well Defined Framework Under process of Development No or Minimal Framework EUROPEAN5 USA5 INDIA2 UNION  Currently there is no clear guidelines  Comparability studies are required and authority for approval of biosimilars  Requires only Phase III clinical to substantiate evidence for safety, trials for 100 patients  No equivalent of ANDA under PH&S act efficacy and quality5 for approval of biosimilars6  Guidelines, including specific clinical CHINA 4  A biosimilar could not be approved until and non-clinical data requirements 12 years after the date on which the for four product types: reference product was first licensed5 Recombinant insulin, human growth factor, erythropoietin and CSFs 6  10-year period for innovator exclusivity, with the opportunity for JAPAN 1 an additional year for new  Pharmaceuticals and medical device indications5 Agency (PMDA ) Japans regulator, expects to finalize a new guideline for the regulation of follow-on Biopharmceuticals this year,  First draft put out for public comment last September CANADA 422 Note: Sources mentioned in the notes section of slide
    23. 23. Biosimilars In the Market Today  Europe - Sandoz –Omnitrope (hGH), Binocrit (“EPO” or erythropoietin); Biopartners -Valtropin (hGH); Hexal –EPO version;  U.S. –Sandoz -Omnitrope  China –EPO versions, Interferons, IL-2, IL-11, GM-CSF, hGHs  India –hGH. EPO, Interferon alpha 2b, insulin  Australia –Omnitrope (Sandoz)  Cuba, Egypt, Africa –EPO versions23
    24. 24. Strategic Options to Tap Biosimilar Market Generics firms enter successfully into the biosimilars market Pharmaceutical companies expand their Biopharmceuticals business and enter biosimilars market opportunistically New types of cooperation between Pharma, Biotech or Generics24
    25. 25. Biosimilars successful if all hurdles passed25
    26. 26. The other side of the Biopharmceuticals……. Safety issue …………??????????  Biologic drugs are orders of magnitude more complex than small molecule drugs  Safety & efficacy of final product is exceptionally sensitive to small changes in manufacturing process  It is difficult to impossible to predict the effect of these small changes— experience counts  Potential for dramatic negative health consequences26
    27. 27. Key Success Factors for Biosimilars 1. Consistent long term strategy 2. Healthy financial structure 3. Comprehensive competitive intelligence 4. Core competencies for manufacturing process 5. Deep clinical development and regulatory expertise 6. Effective marketing & sales skills27
    28. 28. Regulation of Biologics
    29. 29. Challenges in front of Biopharmceuticals From Industry perspective  Long and costly clinical trials  Efficacy  Difference in Sero – Prevalence (Virus etc) and Genetic makeup (Human beings)  Low cost advantage (due to costly raw materials, equipments and labor)  Difficult to copy (Standardization process)29
    30. 30. Continue… From Govt. perspective  No guidelines (Since Biopharmceuticals are in Nascent stage) From Patients perspective  Adverse - effects30
    31. 31. Future of Biopharmceuticals  More diseases will come under the treatment  Effective manufacturing31
    32. 32. Conclusion  Biopharmceuticals or PERISH!!!!!!!  Traditional drug1st generation Biopharmceuticals (small molecules)2nd Generation Biopharmceuticals (large molecules) Follow on Biopharmceuticals Biobetters ….….What next???32
    33. 33. 33

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