Non Clinical Development for ATMP: In Vivo Study Requirements and Strategy in Absence of Relevant Models. Swissmedic

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1 Non Clinical Development for ATMP: In Vivo Study Requirements and Strategy in Absence of Relevant Models Swissmedic

2 Overview 1. Swiss Regulatory Basis for GT/GMO Products and TpPs 2. General Strategies for Preclinical Evaluation during Product Development 3. Strategy in the Absence of Relevant Models 4. Conclusions

3 Overview 1. Swiss Regulatory Basis for GT/GMO Products and TpPs 2. General Strategies for Preclinical Evaluation during Product Development 3. Strategy in the Absence of Relevant Models 4. Conclusions

4 1. Swiss Regulatory Basis In Switzerland ATMP = GT / GMO / TpP products Legal basis (primarily): - Law on Transplantation (TG, SR ) - Law on Therapeutic Products (HMG, SR ) Guidelines (Swiss Guidelines, ICH, EMA) Information on Swissmedic website: Transplantatprodukte: Klinische Versuche GT/GVO: 4

5 Overview 1. Swiss Regulatory Basis for GT/GMO Products and TpPs 2. General Strategies for Preclinical Evaluation during Product Development 3. Strategy in the Absence of Relevant Models 4. Conclusions

6 2. Strategies for Preclinical Evaluation The following aspects ALWAYS need to be addressed: Proof of concept (PoC) - Efficacy, gene expression, target organs, route, dose, dosing scheme, duration of exposure, follow up Kinetics - Distribution, migration, proliferation, persistence, shedding Toxicology - Local / systemic toxicity, immune reactions, adverse ectopic tissue formation, tumorigenicity

7 2. Strategies for Preclinical Evaluation Preclinical studies support clinical studies Pivotal preclinical studies are performed with an investigational product that is identical to or closely comparable with the product to be administered to humans Aim: in vitro / in vivo evaluation (mechanism of action, distribution / migration / proliferation, safety) The preclinical evaluation is performed based on the risks of the individual products (Risk Assessment)

8 2. Strategies for Preclinical Evaluation Early Development Full Development Launch Ph 0 Ph I Ph IIa Ph IIb Ph III REG/Ph IV 8

9 2. Strategies for Preclinical Evaluation Proof of Concept Choice of an animal model (relevant model, disease model, homologous animal model) Specific studies with the relevant product Demonstration of efficacy (clinical indication!) GMO Vaccines: immunological data Gene therapy: gene expression data 9

10 2. Strategies for Preclinical Evaluation Kinetics Studies in a relevant animal model Data with respect to biodistribution, shedding Data with respect to migration / proliferation (TpP) Data with respect to persistence / accumulation / survival (product dependent) Can be combined with safety studies

11 2. Strategies for Preclinical Evaluation Safety No standardized regulatory toxicity program Safety studies have to be performed under GLP Several aspects combined in well designed studies Evaluation of the tumorigenic potential GMO Vaccines: Evaluation of the immunotoxicity Product is identical to or closely comparable with the product to be administered to humans GLP Ordinance:

12 Overview 1. Swiss Regulatory Basis for GT/GMO Products and TpPs 2. General Strategies for Preclinical Evaluation during Product Development 3. Strategy in the Absence of Relevant Models 4. Conclusions

13 3. Strategy in the Absence of Relevant Models: General There is no product on the market in Switzerland: Swissmedic expects preclinical in vivo data Ask for a scientific advice meeting As soon as there is enough experience with approved products, requirements might be adapted on a case-by-case basis The preclinical evaluation is always a risk-based approach

14 3. Strategy in the Absence of Relevant Models: Proof of concept Absence of in vivo data might be acceptable if: The mechanism of action is well described and sufficient in vitro data are available Sufficient Potency data are available (in the frame of the quality documentation)... In any case, the approach needs to be justified

15 3. Strategy in the Absence of Relevant Models: Kinetics Absence of in vivo data might be acceptable if: Cells are encapsulated and applied for a limited time period Sufficient data with comparable vector systems are available (gene therapy)... In any case, the approach needs to be justified

16 3. Strategy in the Absence of Relevant Models: Safety Reduced in vivo data might be acceptable if: Preclinical or clinical data with a comparable system are available Cells are locally applied and rapidly removed due to allogeneic nature of the cell / tissue product (dependent on indication) In any case, the approach needs to be justified and needs to be discussed with the Regulatory Authority

17 3. Strategy in the Absence of Relevant Models: Safety For in vivo gene therapy you always need: Local and systemic toxicity data For ex vivo gene therapy you always need: Data with respect to insertional mutagenicity See below... For cell / tissue therapy you always need: Local toxicity data Evaluation of teratogenicity / tumorigenicity

18 Overview 1. Swiss Regulatory Basis for GT/GMO Products and TpPs 2. General Strategies for Preclinical Evaluation during Product Development 3. Strategy in the Absence of Relevant Models 4. Conclusions

19 4. Conclusions No ATMP product has been approved by Swissmedic in Switzerland All applications need preclinical in vivo data depending on the developmental stage of the product Reduction in the preclinical evaluation can be foreseen under certain circumstances Ask for Scientific Advice at an early stage of development

20

21 Links Swissmedic: ICH: EMA (Guidelines are taken into consideration by Swissmedic): FDA (Guidelines are taken into consideration by Swissmedic):

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