Australian regulatory guidelines for
prescription medicines
Appendix 21: Medicines produced by genetic
manipulation
June 2004
Therapeutic Goods Administration
About the Therapeutic Goods Administration (TGA)
 The TGA is a division of the Australian Government Department of Health and Ageing, and is
responsible for regulating medicines and medical devices.
 TGA administers the Therapeutic Goods Act 1989 (the Act), applying a risk management
approach designed to ensure therapeutic goods supplied in Australia meet acceptable standards
of quality, safety and efficacy (performance), when necessary.
 The work of the TGA is based on applying scientific and clinical expertise to decision-making, to
ensure that the benefits to consumers outweigh any risks associated with the use of medicines
and medical devices.
 The TGA relies on the public, healthcare professionals and industry to report problems with
medicines or medical devices. TGA investigates reports received by it to determine any
necessary regulatory action.
 To report a problem with a medicine or medical device, please see the information on the TGA
website.
Copyright
© Commonwealth of Australia 2004
This work is copyright. Apart from any use as permitted under the Copyright Act 1968, no part may be reproduced by any
process without prior written permission from the Commonwealth. Requests and inquiries concerning reproduction and rights
should be addressed to the Commonwealth Copyright Administration, Attorney General’s Department, National Circuit, Barton
ACT 2600 or posted at http://www.ag.gov.au/cca
Australian regulatory guidelines for prescription medicines – Appendix 21
June 2004
Page 2 of 6
Therapeutic Goods Administration
Appendix 21: Medicines
produced by genetic
manipulation
These guidelines provide information for Australian sponsors intending to submit applications to
the Therapeutic Goods Administration (TGA) to register medicinal products produced by genetic
manipulation.
Medicinal products produced by genetic manipulation may be categorised into two broad
categories:
 genetically modified organisms (GMOs) which are intended for use as medicinal agents;
 products derived or produced from GMOs (GM products).
Office of the Gene Technology Regulator
In Australia, dealings with genetically modified organisms (for example, research, manufacture,
propagation and importation) are controlled under the Gene Technology Act 2000 by the Gene
Technology Regulator to ensure the protection of the health and safety of people and of the
environment. This includes the control of all dealings with live, viable GMOs, including those
intended for use as medicinal agents.
Sponsors intending to apply to the TGA to use a GMO as a medicinal product (including use in a
clinical trial) are advised to also consult the Office of the Gene Technology Regulator (OGTR) to
determine their obligations under the Gene Technology Act 2000. Further information can be
obtained from the OGTR website1.
Sponsors are further advised that the TGA is required to seek the Gene Technology Regulator’s
advice in relation to applications for the supply of therapeutic goods containing a GM product.
The Gene Technology Act 2000 requires the Gene Technology Regulator to maintain a publicly
accessible record of all dealings in Australia that involve GMOs or GM products (the Record). (The
Record includes information on all GM Products approved for supply in Australia under a number
of Acts, including therapeutic goods containing GM products approved for supply under the
Therapeutic Goods Act 1989.) The information required for the Record can be obtained from the
OGTR website.
Sponsors should indicate in their application to the TGA whether they intend to apply to the Gene
Technology Regulator to have the information required for the Record declared to be confidential
commercial information (CCI).
Gene and Related Therapies Advisory Panel
Gene and Related Therapies Research Advisory Panel (GTRAP) is an advisory committee
established under the Research Committee of the National Health and Medical Research Council
(NHMRC). Its terms of reference encompass gene and related therapies in humans. As a major part
of this brief, all clinical trials that involve the introduction of genetic material into human subjects
1
http://www.ogtr.gov.au
Australian regulatory guidelines for prescription medicines – Appendix 21
June 2004
Page 3 of 6
Therapeutic Goods Administration
must be approved by GTRAP prior to final approval by a Human Research Ethics Committee
(HREC).
GTRAP will provide advice to the NHMRC and HRECs on matters relating to human trials of gene
and related therapies proposed for conduct in Australia. It is also required to maintain a publicly
accessible record of Committees on matters relating to human trials of gene and related therapies
proposed for conduct in Australia. Further information can be obtained from the GTRAP website2.
Recombinant biologicals
Guidance on quality issues for recombinant or biotechnological products is provided in European
Union guidelines. Examples which have been adopted by the TGA are listed below. These
documents may be updated or new guidance documents added from time to time. The latest
versions of these guidelines are available at the TGA website 3:
Production and Quality Control of Medicinal Products Derived by Recombinant DNA Technology.
(http://www.tga.gov.au/pdf/euguide/vol3ab1aen.pdf )
Production and Quality Control of Cytokine Products Derived by Biotechnological Processes.
(http://www.tga.gov.au/pdf/euguide/vol3ab3aen.pdf )
Use of Transgenic Animals in the Manufacture of Biological Medicinal Products for Human Use.
(http://www.tga.gov.au/pdf/euguide/vol3ab7aen.pdf )
Position Statement on the Use of Tumourigenic Cells of Human Origin for the Production of Biological
and Biotechnological Medicinal products.
(http://www.tga.gov.au/pdf/euguide/bwp114300en.pdf )
Note for Guidance on Quality of Biotechnological Products: Viral Safety Evaluation of Biotechnology
Products Derived from Cell Lines of Human or Animal Origin.
(http://www.tga.gov.au/docs/pdf/euguide/ich/029595en.pdf )
Quality of Biotechnological Products: Analysis of the Expression Construct in Cell Lines used for
Production of rDNA Derived Protein Products.
(http://www.tga.gov.au/pdf/euguide/vol3ab2aen.pdf )
Quality of Biotechnological Products: Stability Testing of Biotechnological/Biological Products.
(http://www.tga.gov.au/docs/pdf/euguide/vol3a/3ab5aen.pdf )
Development Pharmaceutics for Biotechnological and Biological Products (Annex to Note for
Guidance on Development Pharmaceutics).
(http://www.tga.gov.au/pdf/euguide/bwp032899en.pdf )
Note for Guidance on Quality of Biotechnological Products: Derivation and Characterisation of Cell
Substrates Used for Production of Biotechnological/Biological Products.
(http://www.tga.gov.au/pdf/euguide/ich029495en.pdf )
Note for Guidance on Specifications: Test Procedures and Acceptance Criteria for
Biotechnological/Biological Products.
(http://www.tga.gov.au/pdf/euguide/ich036596en.pdf )
2
3
http://www.nhmrc.gov.au/research/gtrap.htm
http://www.tga.gov.au/industry/pm-euguidelines-adopted.htm
Australian regulatory guidelines for prescription medicines – Appendix 21
June 2004
Page 4 of 6
Therapeutic Goods Administration
GMOs – medicinal products
GMO-medicinal products may include live vaccines (viral or bacterial), viral vectors and
modified somatic cells. Intending sponsors of GMOs intended for use in gene therapy are
referred to the relevant European Union Guidelines, which may be updated from time to time.
Examples include:
Note for Guidance on the Quality, Preclinical and Clinical Aspects of Gene Transfer Medicinal Products.
CPMP/BWP/3088/99 (http://www.tga.gov.au/pdf/euguide/bwp308899en.pdf )
Biotechnology Guideline 3AB6A - Gene Therapy Product Quality Aspects in the Production of Vectors
and Genetically Modified Somatic Cells. (http://www.tga.gov.au/pdf/euguide/vol3ab6aen.pdf)
Australian regulatory guidelines for prescription medicines – Appendix 21
June 2004
Page 5 of 6
Therapeutic Goods Administration
PO Box 100 Woden ACT 2606 Australia
Email: info@tga.gov.au Phone: 02 6232 8444 Fax: 02 6232 8605
www.tga.gov.au
Reference/Publication #