Description
Several developments have fueled the renewed interest in xenotransplantation- the use of live animal cells, tissues and organs in the treatment or mitigation of human disease. The world-wide, critical shortage of human organs available for transplantation and advances in genetic engineering and in the immunology and biology of organ/tissue rejection have renewed scientists' interest in investigating xenotransplantation as a potentially promising means to treat a wide range of human disorders. This situation is highlighted by the fact that in the United States alone, 13 patients die each day waiting to receive a life-saving transplant to replace a diseased vital organ.
Scope & Applicability
Product Classes
6May meet the definition of a regenerative medicine therapy
Points to Consider in the Manufacture and Testing
GT products within the scope of this guidance include products that mediate their effect by the expression of transferred genetic materials.; The main subject of the S12 guidance document
Statutory authorities applied to these products
development program for drug and biological products
Excluded from the scope of this guidance
Stakeholders
10Responsible for qualifications, training, and trial conduct; Individual responsible for trial conduct and data governance at a site.; May delegate tasks but retains overall responsibility; Person responsible for the conduct of the clinical trial at a trial site; Responsible for trial conduct and participant safety; Responsible for trial conduct, data integrity, and investigational product management.; Individual responsible for trial conduct at a site and informing the institution.; maintaining
Party against whom civil money penalties are sought
Population affected by Nipah-virus infection in Singapore
Population affected by Nipah virus encephalitis
Guidelines for Protecting the Safety and Health of Health-Care Workers
Guidelines to Prevent Simian Immunodeficiency Virus Infection
Personnel providing post-xenotransplantation care or handling animal tissues.; Education of Health Care Workers regarding risks associated with xenotransplantation
May observe the consent process as per 45 CFR 46.109(e).
a person who receives or who undergoes ex vivo exposure to a xenotransplantation product; Patient receiving the xenotransplantation product; The human patient receiving the xenotransplantation product.; links the relevant records of the xenotransplantation product recipient
Assist sponsors in the nonclinical evaluation
Regulatory Context
Attributes
1Retention period for health records beyond transplantation
Identified Hazards
Hazards
10Potential public health risk from xenotransplantation products.
Novel progressive disease in cattle
Distinguishing from human cytomegalovirus for xenotransplantation
Scientific discussion of prion diseases
Novel virus in swine closely related to human hepatitis E
Detection of novel strain in pigs with wasting syndrome
Outbreak of Nipah virus among pig-farmers and abattoir workers
Infection by porcine endogenous retrovirus after islet xenotransplantation; Infection risk in human recipients of porcine islet cell xenografts
Infectious disease issues and xenogeneic infections
Disease of animals that may be transmitted to humans.
Related CFR Sections (6)
- 21CFR312.32§ 312.32 IND safety reporting.
(a) Definitions. The following definitions of terms apply to this section:Read full regulation →
- 21CFR50.25§ 50.25 Elements of informed consent.
(a) Basic elements of informed consent. In seeking informed consent, the following information shall be provided to each subject:Read full regulation →
- 21CFR312.23§ 312.23 IND content and format.
(a) A sponsor who intends to conduct a clinical investigation subject to this part shall submit an “Investigational New Drug Application” (IND) including, in the following order:Read full regulation →
- 21CFR312.50§ 312.50 General responsibilities of sponsors.
Sponsors are responsible for selecting qualified investigators, providing them with the information they need to conduct an investigation properly, ensuring proper monitoring of the investigation(s), ensuring that the investigation(s) is conducted in accordance with the general investigational plan Read full regulation →
- 21CFR312.42§ 312.42 Clinical holds and requests for modification.
(a) General. A clinical hold is an order issued by FDA to the sponsor to delay a proposed clinical investigation or to suspend an ongoing investigation. The clinical hold order may apply to one or more of the investigations covered by an IND. When a proposed study is placed on clinical hold, subjectRead full regulation →
- 21CFR312.3§ 312.3 Definitions and interpretations.
(a) The definitions and interpretations of terms contained in section 201 of the Act apply to those terms when used in this part:Read full regulation →
Related Warning Letters (10)
- 2025-12-23
In Vivo Bioavailability-Bioequivalence Studies – Clinical
Maria A. Carballosa, M.D.
- 2025-12-09
Unapproved New Drug/Unlicensed Biological Product/Biologics License Application (BLA)
Celularity, Inc
- 2025-11-18
Sponsor/Investigator
Verdure Sciences, Inc.
- 2025-09-30
Clinical Investigator (Sponsor)
Pamela K. Den Besten, DDS, MS
- 2025-09-23
Clinical Investigator/Sponsor
Ralph A. DeFronzo, M.D.
- 2025-09-09
Clinical Investigator
Shirish M. Gadgeel, M.D.
- 2025-07-15
Clinical Investigator
Mark J. Savant, M.D
- 2025-07-01
Clinical Investigator
Peter Michael, M.D.
- 2025-06-10
Clinical Investigator (Sponsor)
American Behavioral Research Institute, LLC
- 2025-06-03
Bioresearch Monitoring Program
Amy Lightner, MD
See Also (8)
- Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers (Status: Final)
- Content and Format of Investigational New Drug Applications (INDs) for Phase 1 Studies of Drugs, Including Well-Characterized, Therapeutic, Biotechnology-derived Products: Guidance for Industry (Status: Final)
- Postmarketing Safety Reporting for Human Drug and Biological Products Including Vaccines: Draft Guidance for Industry (Status: Draft)
- Guidance for Industry: Providing Regulatory Submissions to CBER in Electronic Format -- Investigational New Drug Applications (INDs) (PDF) (Status: Final)
- Gingivitis: Development and Evaluation of Drugs for Treatment or Prevention (Status: Draft)
- How to Comply with the Pediatric Research Equity Act (Status: Draft)
- Establishment and Operation of Clinical Trial Data Monitoring Committees: Guidance for Clinical Trial Sponsors (Status: Final)
- Clinical Data Needed to Support the Licensure of Seasonal Inactivated Influenza Vaccines: Guidance for Industry (Status: Final)