UK medicines agency calls for a more technologically neutral approach to the current legal definitions of gene therapy medicinal products.
The Medicines and Healthcare products Regulatory Agency (MHRA) is seeking feedback on its proposal to update to the definition of gene therapy medicinal products (GTMPs).
The revisions are intended to align with current advances in science and manufacturing. The resulting modernised, future-proofed framework is intended to increase clarify for developers of these products.
The range of gene therapy types available has expanded since the definition of GTMPs was first developed in 2007, meaning some of these products do not fit neatly into the categories defined in the existing UK legislation, the Human Medicines Regulations 2012.
Requiring GTMPs to be biological in origin excludes synthetic nucleic acid constructs that function identically to biologically derived therapies”
Requiring GTMPs to be biological in origin excludes synthetic nucleic acid constructs that function identically to biologically derived therapies, MHRA stated.
The current legislation on recombinant nucleic acids also does not align with updates to both emerging gene editing approaches delivered through non-recombinant nucleic acid systems and potential non nucleic acid platforms.
Another misalignment is the requirement that the therapeutic effect of a GTMP must derive directly from the recombinant sequence excludes products where genetic modification provides a crucial safety or manufacturing function. This creates classification uncertainty, inconsistent oversight, and potential barriers for developers, the agency added.
Under the proposed revisions, GTMP classification encompass active substances which contain a recombinant or synthetic nucleic acid moiety that undergoes transcription or translation, as well as active substances with a mechanism of action that involves sequence specific genome editing.
Fundamentally, these changes relate to classification of these products and do not amend the regulatory principles applicable to GTMPs.
Public comment can be provided here until 22 June.
Meanwhile, the US medicines regulator has recently opened feedback relating to the latter, issuing draft guidance on standardised methods for the safety assessment of genome editing therapies. FDA Commissioner Marty Makary, said at the time of the publication that the agency’s recommendations offer “clear, scientifically-grounded recommendations for evaluating off-target editing risks using state-of-the-art sequencing technologies”.
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