European Medicines Agency 1 London, 19 March 2009 2 EMEA/CHMP/GTWP/587488/2007 3 4 COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE 5 (CHMP) 6 REFLECTION PAPER 7 QUALITY, NON-CLINICAL AND CLINICAL ISSUES RELATING SPECIFICALLY TO 8 RECOMBINANT ADENO-ASSOCIATED VIRAL VECTORS 9 DRAFT AGREED BY GENE THERAPY WORKING PARTY (GTWP) January 2009 PRESENTATION TO THE COMMITTEE FOR ADVANCED February 2009 THERAPIES (CAT) AND CHMP ADOPTION BY CHMP FOR RELEASE FOR CONSULTATION March 2009 END OF CONSULTATION (DEADLINE FOR COMMENTS) September 2009 10 Comments should be provided electronically in word format using this template to
[email protected] 11 12 KEYWORDS Adeno-associated virus, self complementary adeno-associated virus, recombinant adeno-associated virus, production systems, quality, non-clinical, clinical, follow-up, tissue tropism, germ-line transmission, environmental risk, immunogenicity, biodistribution, shedding, animal models, persistence, reactivation. 13 7 Westferry Circus, Canary Wharf, London, E14 4HB, UK Tel. (44-20) 7418 8400 Fax (44-20) 7418 8613 E-mail:
[email protected] http://www.emea.europa.eu ©EMEA 2009 Reproduction and/or distribution of this document is authorised for non commercial purposes only provided the EMEA is acknowledged 14 15 REFLECTION PAPER ON QUALITY, NON-CLINICAL AND CLINICAL ISSUES 16 RELATING SPECIFICALLY TO RECOMBINANT ADENO-ASSOCIATED VIRAL 17 VECTORS 18 19 TABLE OF CONTENTS 20 1. INTRODUCTION........................................................................................................................