Calls for gene therapies to be made exempt from GMO legislation in Europe

Advanced therapies should be exempt from Genetically Modified Organism (GMO) legislation in Europe, if the sector wishes to stay competitive, according to a collection of life sciences associations.

In a paper published by the Alliance for Regenerative Medicine (ARM), the European Federation of Pharmaceutical Industries and Associations (EFPIA), and the European Association of Bioindustries (EuropaBio), the groups state that current GMO legislation hurts Europe’s ability to attract clinical trials and delays patient access to transformative medicines.

The paper details the complexities of complying with GMO requirements, how they vary significantly across EU Member States, leading to delays to clinical trials focusing on Advanced Therapy Medicinal Products (ATMP).

ATMPs represent new fields of medicines including cell and gene therapies and offer promising treatments for life-threatening diseases which previously had limited treatment options.

Original GMO legislation in Europe was intended to protect food consumers and the environment, but ATMPs have been affected as an unintended consequence due to how they alter genetic material.

The authors of the paper argue that delays to clinical trials for ATMPs, alongside complex and varying GMO legislation, makes the EU less attractive as a region to conduct clinical trials with cell and gene therapies.

The European Commission has attempted initiatives to facilitate and reduce discrepancies across the EU regarding the application of the GMO requirements, but the authors state it still remains difficult to conduct multicenter clinical trials with ATMPs containing or consisting of GMOs involving several EU Member States.

A study on new genomic techniques and the European Commission’s 2020 Pharmaceutical Strategy for Europe recognise how GMO requirements hinder the conduct of clinical trials, with the strategy in particular calling for GMO legislation to be “fit for purpose” for addressing medicines.

During the pandemic, the European Commission granted a temporary derogation from GMO requirements to investigational Covid-19 medicinal products to accelerate the development of vaccines and treatments. This was thought to decrease the amount of time required to complete clinical trials in Europe. Now, the authors are calling for a similar, but permanent, exemption for gene therapies.

“The European Commission recognised that time was of the essence when lifting GMO requirements for Covid-19 vaccines and treatments,” said Paige Bischoff, ARM’s senior vice president of Global Public Affairs. “Time is also very much of the essence for people with cancer, inherited disorders and other life-threatening conditions. We call on the European Commission to take the same measures for advanced therapies and remove the unnecessary and unintended burden of GMO legislation so patients have timely access to transformative, potentially curative medicines.”

The organisations are calling on the European Commission to put forward a proposal by 2022. Without an exemption for gene therapies, the GMO requirements threaten the region’s competitiveness with other parts of the world where GMO legislation is less complex and cumbersome, the organisations say.

For instance, a 2019 ARM report shows that the number of  ATMP clinical trials in Europe stayed roughly flat over a four-year period (2014-2018) while increasing substantially in North America (+36%) and in Asia (+28%).

"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. “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."

“Freeing the conduct of clinical trials with investigational gene therapies from the heavy EU GMO administrative burden is critical for cutting-edge biotechnology companies,” added Violeta Georgieva, EuropaBio’s Legal Affairs Manager. “The use of CRISPR/Cas9, the latest promising tool in genome editing, can be overshadowed in the EU if developers and regulators are to follow the 2018 ruling of the EU Court of Justice, which puts the controversial GMO label on the Nobel Prize-winning CRISPR technology. Our hopes are set on the European Commission to improve patient access to revolutionary treatments by exempting