Cord Blood Licensure. Session 2C: Advanced Cell Therapies April 11, 2013

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1 Cord Blood Licensure Session 2C: Advanced Cell Therapies April 11, 2013

2 Objectives Describe rationale for cord blood licensure Cite licensure regulations Illustrate a case study of one program s pathway to obtaining licensure

3 FDA proposes draft regulatory approach for autologous & allogeneic UCB under IND leading to BLA Docket 96N-0002 Cord Blood Timeline of FDA s Proposed Regulation FDA announces Proposed Approach to Regulation of Cellular and Tissue-Based Products (Section 361 PHS Act) FDA requests Proposed Standards for Unrelated Allogeneic Peripheral and Placental/UCB HSCP Docket 97N-0497 FDA releases Draft UCB Guidance FDA releases Final UCB Guidance HCT/Ps Establishment Rule 21CFR 1271 Subparts A and B HCT/Ps Donor Suitability Rule 21CFR 1271 Subpart C Public meeting HCT/Ps cgtp Rule 21CFR 1271 Subparts D,E, and F Cord Blood Licensure Workshop, March 2010, Used with permission of Liana Harvath

4 Final FDA Guidance and Draft IND Documents were released Oct Guidance for Industry: Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications Guidance for Industry and FDA Staff: Investigational New Drug Applications (INDs) for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications Within 2 years, all public CBBs must have completed the licensing process High profile process CB is one of the first cellular therapy products to undergo licensing process

5 FDA Regulation Due to the intended use in allogeneic recipients, CB is classified as a biologic drug CB is NOT a blood product or subject to blood rules CBB is NOT a blood bank or TM service Regulated under the more stringent biologic drug (Section 351, Part 1270) cgmp rules (Part 211) licensing and general biologics stds (Part 600, 601, 610) Processing is considered manufacturing CBBs are regulated like the pharmaceutical industry

6 Challenges When Applying cgmp to Cellular Therapies Biological variation of starting product Cells vs. chemicals Lot sizes Single patient lots vs. thousands of tablets Manufacturing Process Controls Many drugs can be terminally sterilized CT rely on aseptic processing Mechanism of action known TNC, CD34, CFU or other?

7 The St. Louis Cord Blood Bank Founded in 1995 by Donna Wall, MD First product banked Jan 1996 IND 7183 submitted Jun 30, 1997 Community program operates in collaboration with > 400 physicians at 29 local hospitals Public cord blood bank 127,000 donations; 28,000 processed 2,250 distributed to 34 countries, >20% international Affiliated with National Marrow Donor Program Member of National Cord Blood Inventory (NCBI) DOD s Radiation Injury Treatment Network (RITN)

8 To BLA or not to BLA

9 Pre-BLA meeting

10 Pre-Submission Activities Pre-BLA Meeting Purpose To uncover any major unresolved problems To identify those studies that the sponsor is relying on to establish the drug s effectiveness To acquaint FDA reviewers with the general information to be submitted in the marketing application (including technical information) To discuss appropriate methods for statistical analysis of the data To discuss the best approach to the presentation and formatting of data in the marketing application Lori Tull, FDA Cord Blood Licensure Workshop, March 2010

11 SLCBB Pre-BLA Meeting Timeline Means of directly contacting the agency Organize ourselves, summarize issues, propose questions April SLCBB request May FDA response May SLCBB submitted meeting package June FDA response to questions June face to face meeting in Rockville July FDA minutes

12 Strict cgmp Control & documentation of manufacturing process PLUS equipment qualification, PM, change control Cleaning procedures & disinfecting agents Doesn t specify facility configuration Closed system validation Justification for lack of classification Environmental monitoring for each lot undergoing processing

13 Reagents / Containers Reagent release Raw material qualification Critical identity and quality testing to be repeated, even with a Certificate of Analysis Containers and closures assess supplier s data and its applicability to your product and process Biocompatibility Toxicity Biological tests

14 Clinical Data Clinical safety data Summary of safety experience Narrative of adverse events Details of clinical outcome analyses Specify diagnoses for which product is indicated Provide evidence of efficacy for each additional diagnosis

15 Miscellaneous Reserve samples & attached segments Stability data to support expiration date Containers Storage conditions Source and number of samples Over history Legacy Inventory

16 Assembling and Filing Application

17 Preparation for Filing Formed Licensure Committee and meet weekly Understand the regs, translate pharma-speak Legacy inventory will not meet licensure requirements Confer with admins to ensure their support Align with mission Recognize need to be aggressive in making required changes; physical plant, environmental monitoring, technical policies, Quality Unit Lack expertise in commercialization Considered but rejected hiring consultants

18 Submission Application prep We re not ready! It s not perfect! Cease speculating Due diligence Oct : Submit application Contact review team Real dialoguing begins Dec : Application filed

19 The site visit

20 Site Visit April April Contract Manufacturing Site Preparation Inservices process, behavior Identified escorts, scribes & topic representatives Sign in sheets for attendance, document requests Assessment Opportunity Disruptive, create OT, reduces productivity Only some reviewers office continues requests

21 Don t underestimate Quality Unit Quality Management: Personnel, Equipment, EM, Document Control SOPs in new language Process Control Validation, Qualification and Audits Compliance with Regulation / Accreditation Review records to ensure that no errors have occurred and that those that do occur are fully investigated Responsible for approving/rejecting procedures/specifications affecting identity, strength, quality, and purity of drug product (HPCs) Alters authority of MD

22 Responses

23 Actions Facility segregation, lockers, upgrade HEPA filtration, positive pressure, pass through windows, PPE Micro lab, drafted validation plan Environmental Monitoring plan room classified Cleaning/Disinfection Plan housekeeping contractor Secure evidence of control with intent to scale back QCS Viable / non-viable particle counts equipment qualification process validation materiels mgmt reagent/supply conformance

24 Actions, cont d Collection & Process Validations Expected endpoints determined from historical experience & guidance Stability of cellular potency, component integrity, product sterility Define appropriate time period Define characteristics & endpoints Establishment of expiration date Loss of potency of active ingredient Label ISBT 128 vs NDC Fully implement, not just 16 digit number

25 Impact Making industry standard resin bottle (antibiotic neutralization) Pace with contract manufacturer Changes impact staff morale Productivity Jan-Jun 2010 = 23.5% Jan-Jun 2011 = 20% Jan-Apr 2012 = 17% (Apr = 14%) Cost creep

26 Approval!

27

28 Conclusions Don t take the process lightly The educational process is bi-directional Integrate with regular job Keep the goal in focus Saving lives everyday

29 Thank you & best wishes from St. Louis!

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