CHANGES TO A GENE THERAPY MEDICINAL PRODUCT: REGULATORY VIEWS

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1 CHANGES TO A GENE THERAPY MEDICINAL PRODUCT: REGULATORY VIEWS Maria Cristina Galli, Ph.D. Istituto Superiore di Sanità Roma, Italy EATRIS ATMP platform chair ISCT 2016 Global Regulatory Perspective Workshop Singapore, May 25, 2016

2 DISCLAIMER I attend this conference as an individual expert The views expressed here are my personal views, and may not be understood or quoted as being made on behalf or reflecting the position of the European Medicines Agency/AIFA or any one of its committees or working parties MCG 2016 slide 2

3 GENE THERAPY MOMENTUM Three gene therapy medicinal products on the EU market Glybera: viral vector for lipoprotein lipase deficiency derived pancreatitis Imlygic: oncolytic virus, GM-CSF coding, for melanoma Strimvelis: genetically modified autologous C34+ cells for ADA-SCID treatment. Recently reported encouraging data in clinical trials for other SCIDs or hemophilia, eye diseases Increased industry interest in the field New tools such as genome editing technology, novel engineered or SIN vectors, ips cells MCG 2016 slide 3

4 CHALLENGES FOR DEVELOPMENT Common to both developers and regulators: how to determine if data available for a given gene therapy product is sufficient to allow clinical trials progression Regulatory help tools: timely interaction with most EU national regulatory agencies EMA scientific advice procedure an European consensus view on a development path acceptable to regulators contribution to reduce waste of time and resources MCG 2016 slide 4

5 MAIN RISKS FOR GTMP Germ line transduction: unacceptable in EU (dir. 2001/20-EU Reg.536/2014) Insertional mutagenesis oncogenesis Replicating viral vector target cell lysis/dissemination/shedding Oncolytic viruses ectopic replication Transgene and/or vector immunogenicity impairment of clinical efficacy/immune-toxicity Transgene disregulated expression toxicity/impairment of clinical efficacy MCG 2016 slide 5

6 GTMP DESIGN Critical step: A clear understanding of GTMP molecular structure and biological characteristics is essential to design (=assess!) appropriate Q/NC/C studies The ideal GTMP contains only sequences/proteins needed to achieve the intended clinical goal The real GTMP contains also other sequences/proteins, heritage of early development construct and derived from production system MCG 2016 slide 6

7 APPROPRIATE GTMP DESIGN SHOULD BALANCE SAFETY WITH SOUGHT CLINICAL EFFECT Deletion of sequences responsible for replication ability Oncolytic viruses: replication designed and shown to be restricted to tumour cells Deletion of sequences responsible for integration ability, but: integrative vectors are needed to transduce stem/progenitor cells ex vivo approach, SIN vectors, cell copy number and MOI as low as transduction efficacy can allow Minimal vector backbone to reduce toxicity MCG 2016 slide 7

8 CHANGES DURING GTMP DEVELOPMENT To obtain improved product characteristics and maximise the efficacy/safety profile 1) Changes to production process: e.g. packaging cell line Comparability as for other medicinal products: ICH topic Q5E 2) Changes to GTMP design: e.g. promoter, viral vector serotype, introduction of tissue specific enhancers, insulators, SIN vector. TRANSGENE CHANGE NEW PRODUCT MCG 2016 slide 8

9 CHANGES TO GTMP DESIGN DURING DEVELOPMENT Reflection paper on design modifications of gene therapy medicinal products during development EMA/CAT/GTWP/44236/2009 Published December 2011 The paper aims at giving insight into the types of studies likely to be required in application dossier to support modification in product design introduced during development MCG 2016 slide 9

10 CHANGES TO GTMP DESIGN DURING DEVELOPMENT promoter, viral vector serotype, introduction of tissue specific enhancers, of insulators, SIN vector. assess the impact of modifications on safety/efficacy profile established with the previous product on a case by case basis additional non-clinical studies e.g. toxicology if transgene expression level is increased or biodistribution if tissue tropism changed Extent of additional studies depend also on stage of development possibly further clinical trials MCG 2016 slide 10

11 CHANGES ARE EXPENSIVE: KNOW YOUR PRODUCT! From the very beginning of development have clear in mind what the clinical product will be Any change in vector may impact on the comparability of product across clinical studies carefully plan changes consider possibility of starting development from the beginning if targeting a rare disease, the number of available patients may not easily allow for additional clinical studies MCG 2016 slide 11

12 WHAT IS EATRIS? EUROPEAN INFRASTRUCTURE FOR TRANSLATIONAL MEDICINE Recognised as ERIC (European Research Infrastructure Consortium) 10 participating countries : CZ, DK, ES, FI, FR, IT, NO, EE, NL, SE Negotiations ongoing with: BE, DE, PT, SI, TK, UK EATRIS coordinates a consortium of 75 European academic & nonprofit research institutions of excellence in translational medicine, available to provide the most qualified and state-of-the-art technologies and expertise MCG 2016 slide 12

13 WHAT DOES EATRIS OFFER? 5 platforms: ATMP, vaccines, small molecules, biomarkers, imaging & tracing Access to complete spectrum of unique, high end infrastructure/ expertise for translational development including: GMP manufacturing/glp laboratories Dedicated non-clinical models, capacity to develop new/tailored models Imaging-enabled drug development capabilities Imaging facilities for in vivo animal as well as clinical studies Biomarker validation, prospective validation of diagnostics Clinical experts, access to patients for highly prevalent and/or rare diseases Regulatory experts to help navigate European system IP/TTO experts MCG 2016 slide 13

14 HOW EATRIS CAN HELP Pool of experts from different EU Members States can give advice on how to efficiently plan development in order to reduce changes how to plan the change the studies to be conducted where to carry out them So do not hesitate to contact EATRIS MCG 2016 slide 14

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