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Welcome to the CMC Strategy Forum Japan 2014On behalf of the CASSS Board of Directors and the CMC Strategy Forum Global Steering Committee,we would like to extend to you a warm welcome to the third meeting of the CMC Strategy Forum Japan2014.We are very pleased that with the strong support from the Pharmaceuticals and Medical Devices Agency(PMDA Japan), as well as the Japan Pharmaceutical Manufacturers Association (JPMA), and with thecontinued sponsorship by CASSS and the support from the United States Food and DrugAdministration, that we are continuing with the CMC Strategy Forum Japan 2014. The Forum willfollow the established model of the CMC Forum series with focus on topics and regulatory updatesrelevant for Japan and Asia and will feature an opening regulatory session that will include presentationsfrom PMDA, FDA, EMA, Health Canada, as well as Asian health authorities. The technical sessionswill include discussions on new technology of antibody engineering with focus on glycan engineeringand also bi-specific antibodies, CMC considerations in accelerated approval, life cycle approach toprocess validation in biopharmaceuticals, as well as a session for regenerative medicines, bringingtogether global and Japan specific case studies.The success of the CMC Strategy Forum Japan will depend on your active participation in discussingand raising issues pertaining to the development of biologics. We encourage you to participate wholeheartedly in the panel discussions that have been designed to stimulate exchange of ideas andinformation.We would like to thank the speakers and the panel members who are giving generously of their time andresources and to you for your attendance. We would also like to acknowledge the generosity of ourprogram partners for the continued support of the Forum series: Astellas Pharma Inc.; Baxter Limited;Biogen Idec; Chugai Pharmaceutical Co., Ltd.; Daiichi Sankyo Co., Ltd.; Eisai Co., Ltd.; F. HoffmannLa Roche Ltd.; Kissei Pharmaceutical Co., Ltd.; Kyowa Hakko Kirin Co., Ltd.; Nippon Kayaku Co.,Ltd.; Sanwa Kagaku Kenkyusho Co., Ltd.; Sumitomo Dainippon Pharma Co., Ltd. and TakedaPharmaceutical Company Limited. We are grateful for the expert management from CASSS and theaudio-visual expertise of Michael Johnston from MJ Audio-Visual Productions. Their experience andguidance in the preparation of this Forum has been invaluable.

ACKNOWLEDGEMENTSCMC STRATEGY FORUM GLOBAL STEERING COMMITTEESiddharth Advant, KemWell Biopharma, USAJohn Dougherty, Eli Lilly and Company, USASteven Kozlowski, CDER, FDA, USAJunichi Koga, Daiichi Sankyo Co., Ltd., JapanRohin Mhatre, Biogen Idec, USAAnthony Mire-Sluis, Amgen Inc., USAWassim Nashabeh, F. Hoffmann-La Roche Ltd., Switzerland (Chair)Ilona Reischl, BASG/AGES, AustriaAnthony Ridgway, Health Canada, CanadaNadine Ritter, Global Biotech Experts, LLC, USAThomas Schreitmüller, F. Hoffmann-La Roche Ltd., SwitzerlandMark Schenerman, MedImmune, USAKarin Sewerin, BioTech Development AB, SwedenJapan Scientific Organizing Committee:Ayako Enokida, Pharmaceuticals and Medical Devices Agency (PMDA)Kimio Esumi, Pharmaceuticals and Medical Devices Agency (PMDA)Futaba Honda, Pharmaceuticals and Medical Devices Agency (PMDA)Yasuhiro Kishioka, Pharmaceuticals and Medical Devices Agency (PMDA)Junichi Koga, Daiichi Sankyo Co., Ltd.In Eui Lee, Chugai Pharmaceutical Co., Ltd.Noriyuki Matsumoto, Japan Pharmaceutical Manufacturers Association (JPMA) (Secretariat)Hisako Ohnishi, Japan Pharmaceutical Manufacturers Association (JPMA) (Secretariat)Daisaku Sato, Pharmaceuticals and Medical Devices Agency (PMDA)Yasushi Shikata, Eisai Co., Ltd.Kazuhisa Uchida, Kyowa Hakko Kirin Co., Ltd.Koji Usui, Pfizer Japan, Inc.Toyohiko Yamamoto, Asahi Kasei Pharma CorporationReiko Yanagihara, Pharmaceuticals and Medical Devices Agency (PMDA)

The Scientific Organizing Committee gratefully acknowledges the pharmaceuticaland biotechnology industry for their generous support of the CMC Strategy ForumJapan 2014.SUSTAINING DIAMOND PROGRAM PARTNERF. Hoffmann – La Roche Ltd.SUSTAINING PLATINUM PROGRAM PARTNERBiogen IdecFORUM PROGRAM PARTNERSAstellas Pharma Inc.Baxter LimitedChugai Pharmaceutical Co., Ltd.Daiichi Sankyo Co., Ltd.Eisai Co., Ltd.Kissei Pharmaceutical Co., Ltd.Kyowa Hakko Kirin Co., Ltd.Nippon Kayaku Co., Ltd.Sanwa Kagaku Kenkyusho Co., Ltd.Sumitomo Dainippon Pharma Co., Ltd.Takeda Pharmaceutical Company Limited

LEADING MEDIA PARTNERSBioProcess InternationalInternational Pharmaceutical QualityMEDIA PARTNERSThe Analytical ScientistBioProcessing JournalGenetic Engineering & Biotechnology NewsThe Medicine MakerThe PathologistseparationsNOW.comTechnology Networks Limited

CMC Strategy Forum Japan 2014Scientific Program SummaryMonday, 8 December 201406:30 – 08:15Buffet Breakfast in Dining Café CAMELLIA (South Wing, 1st Floor)06:30 – 10:00Buffet Breakfast in Dining Café CAMELLIA (South Wing, 1st Floor) (foraccompanying guests)07:15 – 09:00Coffee Service in the Maple Room07:15 – 17:00Registration in the Orchard Foyer08:15 – 08:45CASSS Welcome and Introductory Comments in the Orchard RoomWassim Nashabeh, Genentech, a Member of the Roche GroupCMC Strategy Forum Japan 2014 Welcome and Introductory Comments inthe Orchard RoomTakao Yamori, Pharmaceuticals and Medical Devices Agency (PMDA), JapanRecent Trends in the Regulation of Biopharmaceutical Products in the Orchard RoomSession Chairs: Anthony Ridgway, Health Canada and Yoji Sato, National Institute of Health Sciences08:45 – 09:15PMDA Perspective: Recent Trends in the Regulation of BiopharmaceuticalsDaisaku Sato, Pharmaceuticals and Medical Devices Agency (PMDA), Japan09:15 – 09:45FDA Perspective: Recent Trends in the Regulation of BiopharmaceuticalsMarjorie Shapiro, CDER, FDA, USA09:45 – 10:15European Union Regulatory Updates and Recent Developments forBiotechnology ProductsNiklas Ekman, Finnish Medicines Agency, Finland10:15 – 10:45AM Break in the Maple Room10:45 – 11:15Malaysia Perspective: Recent Trends in the Regulation ofBiopharmaceuticalsArpah Abas, Ministry of Health Malaysia (MHM), Malaysia11:15 – 11:45APEC Biotherapeutic Roadmap Activity: How to Work Together withGlobal InitiativesJeewon Joung, Ministry of Food and Drug Safety (MFDS), Korea

Monday, 8 December continued 11:45 – 12:15Taiwan Regulatory Framework and CMC Requirements for BiotechnologyProductsChurn-Shiouh Gau, Center for Drug Evaluation (CDE), Taiwan12:15 – 13:30Buffet Lunch in the Maple Room13:30 – 14:45Panel Discussion – Questions and AnswersArpah Abas, Ministry of Health Malaysia (MHM), MalaysiaNiklas Ekman, Finnish Medicines Agency, FinlandChurn-Shiouh Gau, Center for Drug Evaluation (CDE), TaiwanJeewon Joung, Ministry of Food and Drug Safety (MFDS), KoreaDaisaku Sato, Pharmaceuticals and Medical Devices Agency (PMDA), JapanMarjorie Shapiro, CDER, FDA, USA14:45 – 15:15PM Break in the Maple RoomAspects of Quality Evaluation and Control Corresponding to the Type of Cell-based Products forRegenerative Medicine in the Orchard RoomSession Chairs: Takao Hayakawa, Kinki University and Kazuhisa Uchida, Kyowa Hakko Kirin Co., Ltd.15:15 – 15:25IntroductionTakao Hayakawa, Kinki University, Japan15:25 – 15:50The European Landscape for Regenerative MedicineMargarida Menezes Ferreira, INFARMED, National Authority of Medicines andHealth Products, Portugal15:50 – 16:15Case Study: Cell Therapy Using Accumulated Cells Launched in Japan OnlyKenichiro Hata, Japan Tissue Engineering Co., Ltd., Japan16:15 – 16:40Case Study: Examples Relating to the Quality Control of Cell-basedProductsYuuki Miyatake, Teijin Pharma Limited, Japan16:40 – 17:40Panel Discussion - Questions and AnswersMargarida Menezes Ferreira, INFARMED, National Authority of Medicines andHealth Products, PortugalKenichiro Hata, Japan Tissue Engineering Co., Ltd., JapanDaisuke Maeda, Pharmaceuticals and Medical Devices Agency (PMDA), JapanYuuki Miyatake, Teijin Pharma Limited, JapanYoji Sato, National Institute of Health Sciences, Japan17:45 – 19:15Networking Reception in the Maple Room19:15Adjourn Day One

Tuesday, 9 December 201406:30 – 08:30Buffet Breakfast in Dining Café CAMELLIA (South Wing, 1st Floor)06:30 – 10:00Buffet Breakfast in Dining Café CAMELLIA (South Wing, 1st Floor) (foraccompanying guests)07:15 – 09:00Coffee Service in the Maple Room07:15 – 17:00Registration in the Orchard FoyerAntibody Engineering Technologies and Products: Current Status and Future Prospects in theOrchard RoomSession Chairs: Niklas Ekman, Finnish Medicines Agency and Nana Kawasaki, National Institute ofHealth Sciences08:15 – 08:40Effective Engineered Antibody FormatsIzumi Kumagai, Tohoku University, Japan08:40 – 09:05Bispecific IgG Antibody against FIXa and FX to Treat Hemophilia AManabu Wada, Chugai Pharmaceutical Co., Ltd., Japan09:05 – 09:30GAZYVA / GAZYVARO - The Success Story of a Glyco-engineeredAntibodyElisabeth Kirchisner, Roche Diagnostics GmbH, Germany09:30 – 10:30Panel Discussion - Questions and AnswersNiklas Ekman, Finnish Medicines Agency, FinlandFutaba Honda, Pharmaceuticals and Medical Devices Agency (PMDA), JapanElisabeth Kirchisner, Roche Diagnostics GmbH, GermanyIzumi Kumagai, Tohoku University, JapanManabu Wada, Chugai Pharmaceutical Co., Ltd., JapanTeruhide Yamaguchi, Pharmaceuticals and Medical Devices Agency (PMDA),Japan10:30 – 11:00AM Break in the Maple Room

Tuesday, 9 December continued Accelerated Developing Programs: Unique CMC Consideration in the Orchard RoomSession Chairs: Junichi Koga, Daiichi Sankyo Co., Ltd. and Wassim Nashabeh, F. Hoffmann-La RocheLtd.11:00 – 11:10IntroductionJunichi Koga, Daiichi Sankyo Co., Ltd., Japan11:10 – 11:35CMC Considerations and Challenges for Accelerated ProgramsYasuhiro Kishioka, Pharmaceuticals and Medical Devices Agency (PMDA),Japan11:35 – 12:00Challenges and Opportunities for Commercial Manufacturing Readiness andLaunch of Breakthrough Therapy ProductsEarl Dye, Genentech, a Member of the Roche Group, USA12:00 – 12:25Risk-based Analytical Life Cycle Steps for Accelerated ProductsStephan Krause, AstraZeneca Biologics, USA12:25 – 13:25Panel Discussion – Questions and AnswersBrigitte Brake, Federal Institute for Drugs and Medical Devices, BfArM,GermanyEarl Dye, Genentech, a Member of the Roche Group, USAYasuhiro Kishioka, Pharmaceuticals and Medical Devices Agency (PMDA),JapanStephan Krause, AstraZeneca Biologics, USAMarjorie Shapiro, CDER, FDA, USAKazuhisa Uchida, Kyowa Hakko Kirin Co., Ltd., Japan13:30 – 14:45Buffet Lunch in the Maple RoomLifecycle Approach to Process Validation in the Orchard RoomSession Chairs: Kowid Ho, F. Hoffmann-La Roche Ltd. and Atsushi Matsumoto, Kyowa Hakko KirinCo., Ltd.14:45 – 15:10PMDA Perspective: Regulatory Updates on Process Validation StandardKazunobu Oyama, Pharmaceuticals and Medical Devices Agency (PMDA),Japan15:10 – 15:35EMA Guidance Documents on Process Validation for Biotechnology-derivedMedicinal Products – A Regulatory UpdateBrigitte Brake, Federal Institute for Drugs and Medical Devices, BfArM,Germany

Tuesday, 9 December 2014 continued 15:35 – 16:00The Lifecycle of Process Validation: An Industry Case Study on ContinuedProcess VerificationStefanie Pluschkell, Pfizer, Inc., USA16:00 – 16:30PM Break in the Maple Room16:30 – 17:30Panel Discussion – Questions and AnswersBrigitte Brake, Federal Institute for Drugs and Medical Devices, BfArM,GermanyJeewon Joung, Ministry of Food and Drug Safety (MFDS), KoreaAtsushi Matsumoto, Kyowa Hakko Kirin Co., Ltd., JapanKazunobu Oyama, Pharmaceuticals and Medical Devices Agency (PMDA),JapanStefanie Pluschkell, Pfizer, Inc., USA17:30 – 18:00CMC Strategy Forum Japan 2014 RecapNadine Ritter, Global Biotech Experts, LLC, USA18:00 – 18:15Closing RemarksWassim Nashabeh, Genentech, a Member of the Roche Group, USA18:15Adjournment

Welcome and Introductory CommentsTakao YamoriPharmaceuticals and Medical Devices Agency (PMDA), JapanNOTES:

Recent Trends in the Regulation of Biopharmaceutical ProductsSession Chairs: Anthony Ridgway, Health Canada and Yoji Sato, National Institute of Health SciencesIn this opening scientific session, an international group of regulators representing the US FDA, EMA,MHM, MFDS, CDE and PMDA, will present their views on recent trends in the regulation ofbiopharmaceutical products. Within this general category, presenters will be asked to includeinformation that will contribute to panel discussions covering three themes: Innovative regulatory strategies for enabling the rapid development and the potential for earlymarketing approval of highly promising new biotherapeutic products;Areas of frequent consultations between industry and regulators, including, if applicable, cell andgene therapy, and biosimilars;International regulatory convergence and, in particular, how the picture for Asia might appeardifferent than the global picture.NOTES:

Presenter’s Abstracts and PresentationsPMDA Perspective: Recent Trends in the Regulation of BiopharmaceuticalsDaisaku SatoPharmaceuticals and Medical Devices Agency, JapanThe Japanese regulation has improved its review performance over the last decade. In the current midterm plan starting from 2014, PMDA will further shorten its review period with increasing staff size. Inthis presentation, we will discuss the current challenges of CMC review for biologics including postapproval changes. In addition, biosimilars update will be also touched with a focus on the issuessurrounding global developments such as the choice of reference product and regional clinical data.We will also outline Japanese new legislations for regenerative medicine (cellular and tissue-basedproducts) enacted on 25 November 2014. It enables early patient access to promising therapies, usingconditional and time-limited approval scheme in place for regenerative medical product review. At thesame time, for all pharmaceuticals including biopharmaceuticals, “Forerunner package strategy” hasbeen introduced to implement “accelerated approval” (including rolling submission). Such early accessschemes would raise the issues of quality evaluation and validation during the development, due to thelimited experience of batches and timeline for regulatory process. We will take an appropriate productlifecycle approach in the post-approval changes and validation and will step further global collaborationamong regulators and with industries as well.NOTES:

FDA Perspective: Recent Trends in the Regulation of BiopharmaceuticalsMarjorie ShapiroCDER, FDA, Silver Spring, MD USABiopharmaceuticals are among the most successful drugs on the market and represent an increasingproportion of drugs in development. The quality of these products is paramount and decisions should bemade with patient needs in mind. Initiatives such as the reorganization of quality review functions atCDER, the development of biosimilar products, breakthrough designation, the lifecycle approach toprocess validation and challenges in understanding the impact of antibody engineering will be presented.NOTES:

European Union Regulatory Updates and Recent Developments for Biotechnology ProductsNiklas EkmanFinnish Medicines Agency, FinlandThis presentation will provide an update on a number of topics which important for developingbiopharmaceuticals. The new clinical trial regulation published in the Official Journal of the EuropeanUnion in May 2014, will change the way clinical trials are authorized in the EU. The main goal of theregulation is to harmonize the regulatory requirements, provide high standard for patient safety, andcreate an environment that is favourable for conducting clinical trials. The new regulation will alsoincrease the transparency of clinical trial data. The principles of transparency in decision making will befurther strengthened by the recent decision from the EMA Management Board to publish clinical reportsstarting from 1 January 2015. In addition to the discussion on the clinical trials regulation and the EUtransparency policy, the presentation will also summarize the current status of the EU pilot project onadaptive licensing, as well as the future directions in the field of Advanced Therapy Medicinal Products(ATMPs) based on the recent European Commission report on the ATMP regulation. Furthermore, themain activities of the CHMP Biologicals Working Party (BWP) during 2014 will be reviewed.NOTES:

Malaysia Perspective: Recent Trends in the Regulation of BiopharmaceuticalsArpah AbasMinistry of Health Malaysia (MHM), MalaysiaBiotechnology medicines hold some of the greatest promise for medical breakthroughs. There is agrowing interest in making biosimilars, which have the potential to lead to enormous cost-savings inhealthcare without reducing the level of care to patients.Biosimilar regulatory pathway is a paradigm shift that requires demonstration of high similarity in termsof quality, safety and efficacy to the reference product. However, worldwide situation shows that thepathway is implemented rather non-homogenous particularly in developing countries. The World HealthAssembly in May 2014 passed a resolution to develop the necessary scientifically-based regulatoryframeworks that promote access to products that are affordable, safe, efficacious and quality, taking noteof the relevant of WHO guidelines on similar biotherapeutic products (SBPs).The next wave of biosimilars are monoclonal antibodies (mAbs) that have great potential for clinicaluse. As such there is much to learn and more challenges wait.The field of cell and gene therapy products (CGTPs) is radically changing through the use ofbiotechnology and products are moving towards licensure. Due to their unique, diverse and complexmanufacturing and the poor fit into the biologics pathway, a new framework is necessary to ensurepublic safety but presents numerous challenges.Malaysia had the draft CGTPs guideline completed and on circulation now for comments. Regulation ofCGTPs is still evolving, as befits a relatively young developing field. As reforms move forward,worldwide regulatory convergence and sharing knowledge and experience will be vital to effectiveregulation, since safety issues have no borders. The future success of biosimilars and CGTPs depends onsound science and putting patients first. Science, even with broad-band, takes time and due diligence.NOTES:

APEC Biotherapeutic Roadmap Activity: How to Work Together with Global InitiativesJeewon JoungMinistry of Food and Drug Safety (MFDS), KoreaNOTES:

Taiwan Regulatory Framework and CMC Requirements for Biotechnology ProductsChurn-Shiouh GauCenter for Drug Evaluation (CDE), TaiwanAbstract and slides were not available at the time of printing.NOTES:

Recent Trends in the Regulation of Biopharmaceutical ProductsPlenary SessionPanel Discussion – Questions and AnswersArpah Abas, Ministry of Health Malaysia (MHM), MalaysiaNiklas Ekman, Finnish Medicines Agency, FinlandChurn-Shiouh Gau, Center for Drug Evaluation (CDE), TaiwanJeewon Joung, Ministry of Food and Drug Safety (MFDS), KoreaDaisaku Sato, Pharmaceuticals and Medical Devices Agency (PMDA), JapanMarjorie Shapiro, CDER, FDA, USAThe following questions will guide the panel discussion:Theme 1) - Innovative regulatory strategies for enabling the rapid development and the potential forearly marketing approval of highly promising new biotherapeutic products: What regulatory frameworks and pathways are available for the potential accelerated approval ofpromising new products? What are the advantages and disadvantages of different approaches?How will regulators in emerging markets address these concepts?In some regulatory jurisdictions, evaluations of marketing applications may be streamlined ifapprovals have been made in certain “benchmark” regulatory agencies. How will“breakthrough”-type approaches and accelerated approval pathways, with their reliance on lesswell-developed data be accommodated?Theme 2) - Areas of frequent consultations between industry and regulators, including, if ap