Cellular and Gene Therapy Products - CBER Update
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1 DEPARTMENT OF HEALTH AND HUMAN SERVICES CENTER FOR BIOLOGICS EVALUATION and RESEARCH Cellular and Gene Therapy Products - CBER Update Well Characterized Biological Products January 12-14, 2009 Kimberly Benton, Ph.D. Deputy Director Division of Cellular and Gene Therapies FDA/CBER/OCTGT
2 Cellular and Gene Therapies and Related Products (1) Stem cells and stem cell-derived products (hematopoietic, mesenchymal, embryonic, umbilical cord blood, etc) Cancer Vaccines and immunotherapies Dendritic cells, activated T lymphocytes (TIL, LAK), B cells, monocytes, cancer cells chemically modified or unmodified Peptides, recombinant proteins Somatic cells Allogeneic pancreatic islets, Chondrocytes, myoblasts, keratinocytes, hepatocytes Cell lysates and extracts Cells plus scaffold/matrix Encapsulated cells Tissue engineering construct
3 Cellular and Gene Therapies and Plasmids Viral vectors Adenoviruses Related Products (2) Adeno-associated Virus (AAV) Retroviruses Herpes Simplex Virus Pox Viruses (Vaccinia, Fowlpox, Canary pox) Bacterial Vectors (Listeria, Salmonella, etc) Oncolytic viruses Gene modified cells
4 Total Active Files in OCTGT (IND, IDE, MF) FY
5 New IND Applications FY Cell Therapy Gene Therapy
6 IND Amendments FY
7 New IND Applications Commercial or Research Sponsors commercial research
8 Trends in Cell Therapy Sources of adult stem cells Placental and amniotic membrane, adipose Cell Products designed to induce immune tolerance Xenotransplantation Cells encapsulated in a biomaterial Tissue engineering
9 Trends in Gene Therapy AAV Vectors Increased use of serotypes other than AAV-2 AAV-1, AAV-8, AAV-1/2, potential use of AAV-5, AAV-6 Self complementary vectors (SC-AAV) Use of AD-5/35 vectors Increased use of HSV Vectors Replicating vectors Oncolytic viruses Bacterial vectors
10 RNA AAV Poxvirus Retrovirus Adenovirus Plasmids Active Gene Therapy INDs as of May 2008
11 Challenges Small lot size/limited sample volume Limited shelf life (due to cell viability) Patient to patient variability and cellular heterogeneity No terminal sterilization Ancillary materials- limited sources, quality Lack of reference standards Complex manufacturing schemes Multiple potential mechanisms of action Control of cell differentiation Storage/shipping conditions Biocompatibility with biomaterials or delivery device
12 Regulatory challenges Most regulations not written with cell and gene therapy in mind Limited success stories (i.e. licensed products) to learn from or use as examples Experience with other biologics such as blood, vaccines or therapeutic proteins and antibodies may not be directly applicable
13 Common Areas of Need Starting cells or cell banks, ancillary materials Safe, consistent quality and activity Availability Bioprocessing Manufacturing that is transferable to commercial operation and scale Assays Product Release and characterization, stability and comparability protocols capacity to detect product degradation capacity to detect and assess effects of changes Reference Standards
14 Outreach to Cell and Gene Therapy Stakeholders Advisory Committees Regulations Guidance Documents Workshops Liaison Meetings International Harmonization
15 Safety of Cell Therapies Derived from Human Embryonic Stem Cells CTGT Advisory Committee April 10, 2008 Safety Concerns Product Characterization Trial Design
16 CTGTAC April 10, 2008 Safety Concerns Stem cells and inappropriate differentiation Teratoma Ectopic tissue Currently concerns restrict direct use of hesc Persistence of Undifferentiated Cells Likely to be present in ESC-derived products Proliferation, migration Anatomic location and constraints Enclosed space (eg IC vs. IV administration)
17 CTGTAC April 10, 2008 Product Characterization Establish sensitive analytical methods to detect cells with undesired characteristics Minimize undifferentiated stem cells Identify characteristics capable of reliably predicting safety and anticipating clinical effectiveness in-process and lot release testing Ensure that products are as safe as possible current limitations in scientific knowledge
18 CTGTAC April 10, 2008 Major Considerations Stronger than usual proof of concept evidence may be required The dose of cells administered to humans should be below the minimum number of cells observed to form tumors in animal models First in man clinical applications should be picked carefully due to inherent risks Long term follow up recommended due to perceived risk
19 Regulations/Guidance Guidance for Industry: CGMP for Phase 1 Investigational Drugs 7/15/2008 Current Good Manufacturing Practice and investigations New Drugs Intended for Use in Clinical Trials; Final Rule 7/15/2008
20 Guidance Draft Guidance for Industry: Validation of Growth-Based Rapid Microbiological Methods for Sterility Testing of Cellular and Gene Therapy Products 2/11/2008 Guidance for Industry: Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Somatic Cell Therapy Investigational New Drug Applications (INDs) 4/9/2008 Guidance for Industry: Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs) 4/9/2008 Draft Guidance for Industry: Considerations for Allogeneic Pancreatic Islet Cell Products 5/21/2008
21 Guidance Draft Guidance for Industry: Potency Tests for Cellular and Gene Therapy Products (October 2008) Provides manufacturers of cellular and gene therapy (CGT) products, with recommendations for developing tests to measure potency For IND and BLA stages Does not make recommendations regarding specific types of potency assays, nor does it propose criteria for product release.
22 Meetings/Workshops Animal Models for the Treatment of Acute Radiation Syndrome September 17-18, 2008 FDA/NIST Sponsored Workshop In Vitro Analyses of Cell Scaffold Products December 6-7, 2007 FDA Public Workshop: Processing of Orthopedic, Cardiovascular, and Skin Allografts October 11-12,
23 International Harmonization Activities ICH Gene Therapy ICH Considerations General Principles to Address the Risk of Inadvertent Germline Integration of Gene Therapy Vectors, published 10/2006 Oncolytic Viruses, published 11/2008 Viral/Vector Shedding, target completion 6/2009 ICH Guidance Proposing to further develop Viral/Vector shedding considerations to formal guidance Input from US stakeholders FDA-EMEA Cluster on Advanced Therapy Medicinal Products
24 Contact Information Kimberly Benton, PhD General CBER Issues Office of Communication, Outreach and Development (OCOD) (Formerly called Office of Communication, Training and Manufacturers Assistance) Telephone: or Consumers Health Care Professionals: Manufacturers Regulated Industry: INTERNET: CBER Regulatory and Guidance Documents:
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