Why does the regulation of RNA products need to evolve?
RNAs, which, like DNA, are another facet of genetic therapies, are becoming increasingly important in the therapeutic arsenal. Many RNA-based products (drugs and vaccines) are now under development in many therapeutic areas (cancers, genetic diseases, infectious diseases, cardiovascular diseases, metabolic diseases, etc.) or are already on the market (casimersen, elasomeran, eterplirsen, lumasiran, nusinersen, patisiran, tozinameran, etc.).
These products include both coding RNAs that will allow the production of proteins, mRNAs, and non-coding RNAs, which, most often, will intervene in the regulation of protein production (antisense oligonucleotides, aptamers, small interfering RNAs, micro RNAs, activating RNAs, guide RNAs).
In an article published in Frontiers in Medicine, two researchers from the University of Burgundy, Mathieu Guerriaud from the Centre de Recherche sur le Droit des Marchés et des Investissements Internationaux (CREDIMI) and Evelyne Kohli from the Laboratoire d'Excellence LipSTIC describe the classification that European legislation applies to the various RNA-based products. These will be classified according to their target for vaccines or according to their production mode (chemical or biological) for drugs. In the face of technological developments, their analysis highlights the problems raised by the heterogeneity of their status and the need to clarify the definition of a biological medicinal product and a recombinant nucleic acid in a context of lack of international harmonization, particularly between the EU and the United States. In particular, the authors emphasize that the upcoming revision of the European regulations opens a window of opportunity for the implementation of specific recommendations for RNA-based drugs in terms of authorization, production, toxicological studies and pharmacovigilance.
Référence : Guerriaud, M. and E. Kohli, RNA-based drugs and regulation: Toward a necessary evolution of the definitions issued from the European union legislation. Frontiers in Medicine, 2022. 9. https://doi.org/10.3389/fmed.2022.1012497