Stem Cell Therapy A Regulatory Approach By: Gary Yingling and Karl Nobert gary.yingling@klgates.com and karl.nobert@klgates.com October 7, 2008
What is a stem cell? � “Serving as a sort of repair system for the body, they can theoretically divide without limit to replenish other cells for as long as the person or animal is still alive. When a stem cell divides, each ‘daughter’ cell has the potential to either remain a stem cell or become another type of cell with a more specialized function, such as a muscle cell, a red blood cell, or a brain cell.” - Stem Cell Information, National Institutes of Health � All stem cells – regardless of source – share 3 general properties: � Capable of dividing and renewing themselves for long periods of time � Initially unspecialized � Can give rise to specialized cell types - Stem Cell Overview, National Institutes of Health 1
Statutory Authority � Public Health Service Act (“PHS”) � “The Surgeon General, with the approval of the Secretary, is authorized to make and enforce such regulations as in his judgment are necessary to prevent the introduction, transmission, or spread of communicable diseases…” Section 361, 42 U.S.C. § 264. � Biologics – Section 351, 42 U.S.C. § 262. � Federal Food, Drug, and Cosmetic Act (“FDC Act”) � Drugs – Sections 501-528 (Chap. V), 21 U.S.C. §§ 351- 360dd. � Devices – Sections 501-528 (Chap. V), 21 U.S.C. §§ 351- 360dd. 2
Regulatory Authority � Regulatory Status (HCT/P only, biologic, drug or device) � Dependent on a product’s formulation, preparation and intended use � Human Cells, Tissues, and Cellular and Tissue-Based Products (“HCT/Ps”) � 21 C.F.R. Part 1271 � Biologics � 21 C.F.R. Pts. 600-680 � Drugs � Drugs (General) 21 C.F.R. Pts. 200-299 and Drugs for Human Use 21 C.F.R. Pts. 300-460 � Devices � 21 C.F.R. Pts. 800-898 3
Regulatory Authority (continued) � Regardless of regulatory status: � Registration and Listing Requirements � Donor Eligibility Requirements � Adverse Event Reporting � Requirements to prevent the introduction, transmission or spread of communicable diseases 4
Criteria Considered for Determining the Regulatory Status of a Stem Cell Product � Degree of manipulation � Homologous use v. Non-Homologous Use � Combined with another article � Systemic effect and/or dependence on metabolic activity (autologous use? allogeneic use in a first- degree or second-degree relative? reproductive use?) 5
Marketing Option #1 For stem cell products that meet the criteria in 21 C.F.R. § 1271.10: � Only minimally manipulated � Homologous use � Not combined with another article (with limited exceptions) � Either (i) the product does not have a systemic effect and is not dependent upon metabolic activity of living cells for its primary function; or (ii) the product has a systemic effect or is dependent upon metabolic activity for its primary function, and is for autologous, allogeneic or reproductive use If yes, regulated solely under PHS sec. 361 and 21 CFR Part 1271 6
Marketing Option # 1 Requirements: � No pre-market review or approval � Comply with cGTPs (intended to prevent the introduction, transmission, or spread of communicable disease) � Reporting / Labeling � Inspections / Enforcement 7
Marketing Option #2 For stem cell products that exceed the criteria in 21 C.F.R. § 1271.10: � More than minimally manipulated � Intended for non-homologous use � Combined with a non-cellular or non-tissue component � Either (i) systemic effect and dependent on metabolic activity for its primary function, or (ii) systemic effect or dependent on metabolic activity for primary function, and for autologous, allogeneic or reproductive use. If yes, regulated as a drug, device and/or biological product under the FDC Act and/or Section 351 of the PHS Act. 8
Marketing Option # 2 � Biologics � Investigational New Drug (IND) Application / Biologic License Application (BLA) � PHS Section 351 � Manufacturer must show that the cellular or tissue product is safe, pure, and potent and that the facility in which the product is manufactured, processed, packed, or held meets established quality control standards. (42 U.S.C. § 262(a)) 9
Marketing Option #2 (continued) � Drugs � IND / New Drug Application (NDA) � Requires pre-market review and approval � Must show that the product is safe and effective � Compliance with Current Good Manufacturing Practices (cGMPs) � “Well Characterized” � User Fees � Devices � Investigational Device Exemption (IDE) / 510(k) or Pre-Market Approval � Requires pre-market clearance or approval � Compliance with Quality System Regulations (QSR) � User Fees 10
FDA Issues Warning Letter � Warning Letter (July 25, 2008) � Stem cell therapy being marketed as a drug (“for treatment of painful and debilitating orthopedic conditions”) � Marketed therapy exceeds established criteria in 21 C.F.R. § 1271.10 � Requires a BLA and IND 11
Veterinary Stem Cell Therapy – Regulatory Overview The Therapy: Practice of Veterinary Medicine The Product: 1) USDA 2) FDA � Currently, no specific regulations governing the practice 12
Veterinary Stem Cell Therapy – Current Regulation Biologics � USDA � The Virus, Serum, and Toxin Act of 1913 (“VSTA”) � Interstate shipment � Registration and listing requirements � U.S. Veterinary Biologics Establishment License � U.S. Veterinary Biologics Products License 13
Veterinary Stem Cell Therapy – Current Regulation (continued) � Drug � FDA � Food, Drug, and Cosmetic Act � INADA � NADA—requires study data supporting the safety and effectiveness of the product 14
Questions? Gary L. Yingling Karl M. Nobert gary.yingling@klgates.com karl.nobert@klgates.com (202) 778-9124 202-778-9460 15
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