EMA adopts guideline on requirements for clinical-stage ATMPs
The European Medicines Agency (EMA) has recently adopted a guideline outlining the quality, non-clinical, and clinical requirements to support a clinical trial application for investigational advanced therapy medicinal products (ATMPs).The guideline revises a draft version released for public consultation in March 2024. (RELATED: EMA revises draft guideline on data to support clinical trials for ATMPs, Regulatory Focus 25 March 2024).
The multidisciplinary guideline covers the development of gene therapy medicinal products, somatic cell therapy medicinal products, tissue engineered products, and combined ATMPs.
EMA made various updates to the guidelines based on public feedback, as noted in an overview summary of the responses.
One change was the inclusion of a reference to the International Council for Harmonisation’s (ICH) E11 guidance, which addresses the clinical investigation of medicinal products in the pediatric population for ATMPs.
EMA also stated that the guideline will eventually be updated to include more information on developing gene editing products when more experience is gained in this area, in response to a request for additional direction.
A new reference has been added regarding device-drug combination ATMPs in the introduction. The updated text states that “combined ATMPs, which incorporate materials classified as medical devices as an integral part of the product, include one or more medical devices (Regulation (EC) No 1394/2007, Art. 2 (d)). This combination can apply to both cell-based and gene therapy medicinal products.”
Additionally, new text in the introduction now encourages product developers to seek early guidance at the national or European level to inform product development.
EMA said that sponsors should adopt a risk-based approach when evaluating the quality, non-clinical, and clinical data for these products. This risk assessment should consider the origin of the cells, the type of vector or method used for genetic modification, the manufacturing process, and the specific therapeutic use of the product.
The guideline notes that “an immature quality development may compromise the use of the clinical trial data in the context of a marketing authorisation application (e.g. if the product has not been adequately characterised). A weak quality system may also compromise the approval of the clinical trial if deficiencies are apparent from the submission that pose a risk on the safety of trial subjects and the robustness of data.”
The guideline will go into effect on 1 July 2025.
Guideline, Overview of Comments
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