ATMP trade organizations call for GMO exemption in EU

Regulatory NewsRegulatory News | 27 May 2021 |  By 

In a joint paper and press release, major trade associations for cell and gene therapy developers are calling for relaxation of EU regulations that treat these advanced therapies as genetically modified organisms (GMOs).
The Alliance for Regenerative Medicine (ARM), European Federation of Pharmaceutical Industries and Associations (EFPIA), and the European Association of Bioindustries (EuropaBio) joined in the 24 May publication of the paper in Human Gene Therapy, issuing a press release the next day. Both the paper and the statement call for advanced therapies and medicinal products (ATMPs) to be exempted from GMO legislation.
“Gene therapies based on genome editing techniques are currently considered to be GMOs in the EU,” noted the authors. Applying EU’s GMO legislation to investigational, cutting-edge cell and gene therapies has slowed the progress of gene therapy in the EU, claimed the authors, citing 2019 data from an ARM report. That report shows Europe lagging far behind North America and Asia in the number of ATMP clinical trials in each region.
“Having insufficient clinical development of advanced therapies in Europe is a missed opportunity and a major issue that needs to be addressed, with patients having limited early access to these therapies, physicians having less experience with the products when they come to market and marketing authorization applications with limited data on EU patients,” wrote the authors.
The EU’s “complex and cumbersome” handling of investigational medicines stands in contrast with how the US Food and Drug Administration (FDA) has dealt with the issue, according to the paper. Since 2015, FDA guidance has recommended that, in most cases, GMO environmental risk assessments are not needed for gene therapies or for vaccines or recombinant products that use viruses or microbial products.
Currently, the EU approach is constrained by a 2018 ruling from the Court of Justice that “organisms obtained by the new techniques of directed mutagenesis are considered GMOs and are, therefore, subject to the obligations laid down by the GMO directive,” explained the authors. Two directives, 2001/18/EC and 2009/41/EC, now set the framework for handling GMOs in the EU member states. However, since 2018, scientific advisors to the EC have been wrestling with the implications of the application of these directives to ATMPs.
The paper outlines efforts made in the last several years to harmonize the approach among member nations, since these differences were leading to delays in the progress of clinical trials. A joint action plan has borne some fruit, including the generation of a common application form and good practice document for each type of product that falls under GMO regulations.
However, efforts to date are still “inadequate,” according to the paper, which summarizes a 2020 survey of ATMP developers. That survey found substantial roadblocks, with many respondents reporting that “the preparation and submission of the GMO package and the implementation at the clinical sites remain very time consuming and resource intensive.”
The public health emergency of the COVID-19 pandemic spurred more thought on the matter. In June 2020, the EC proposed relaxing GMO regulation requirements for COVID-19 vaccines and therapeutics. The Commission granted these investigational products a temporary derogation from the GMO regulations, with GMO compliance delayed until full marketing authorization is sought. (RELATED: European Commission proposes relaxing GMO regs for COVID-19 vaccines, Regulatory Focus 18 June 2020)
"In 2020, we welcomed the derogation from GMO legislation for COVID-19 treatments or vaccines in clinical trials,” said Pär Tellner, Director of Regulatory Affairs at EFPIA, in the press statement. “Member companies are increasingly reporting how the derogation has removed the significant and time-consuming hurdles associated with GMO submissions, in addition to the clinical trial application. Swift action to a permanent exemption from GMO legislation allows the EU to prosper and most importantly for patients to continue to receive transformative, potentially life-saving therapies."
The time has come to expand and make permanent this change, say the ATMP developers. “After more than 30 years of scientific progress in the development of safer gene therapy vectors and experience based on numerous clinical gene therapy trials worldwide, we can now conclude with confidence that the environmental risks associated with this therapy are negligible,” wrote the authors, calling for a legislative proposal to amend or supplement the current GMO framework.
ARM et al., Human Gene Therapy


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Tags: ATMPs, EMA, EU, GMOs

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