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  • 1.  Shedding data to satisfy EMA

    This message was posted by a user wishing to remain anonymous
    Posted 09-26-2025 14:59
    This message was posted by a user wishing to remain anonymous

    What is an acceptable number of clinical samples to test for shedding following ocular administration of an AAV GTx, to support filing in EU?

    Does it need to be a % of the treated participants, or a sufficient number of participants independent of the size of the clinical study?

    If anyone has that "magic" number, please let me know; with the understanding that everything is case-by-case.

    Thanks, Gracias, Merci, Danke, Grazie, Obrigado, ありがとう, Kiitos, شكراً,....



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  • 2.  RE: Shedding data to satisfy EMA

    Posted 10-07-2025 17:43

    Using 

    • EMA Guideline on the quality, non-clinical and clinical aspects of gene therapy medicinal products (EMA/CAT/80183/2014)
    • EMA Reflection Paper on Management of Clinical Risks Derived from Shedding of Viral Vectors (EMEA/CHMP/GTWP/125459/06)

    Where

    • Shedding studies should be performed in a sufficient number of patients to characterize the pattern, magnitude, and duration of shedding.
    • The number of subjects and samples must be justified scientifically, considering the route of administration, vector dose, and patient population size.

    And thus, the best/suggested practice to Support EU Filing

    • 6–12 patients in early clinical trials (FIM or Phase I/II) is generally acceptable for shedding assessment following ocular AAV administration.
    • Ensure longitudinal sampling (multiple time points per patient).
    • Provide a scientific justification in the IMPD and ERA, explaining why this number is sufficient (e.g., ocular compartmentalization, limited vector dose, prior data).
    • If systemic exposure is plausible (e.g., via nasolacrimal drainage), regulators may request data from upper respiratory samples as well.
    • As always, consult/share with EMA about these suggestions.



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    Robert Falcone Ph.D., FTOPRA, FRAPS
    Global Manager
    Integrated DNA Technologies
    Little Falls NJ
    [email protected]

    Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
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