Environmental risk assessment of advanced therapies containing genetically modified organisms in the EU

Abstract

Gene therapy medicinal products have the potential to provide curative treatment to many diseases with current limited therapeutic options. As advanced therapy medicinal products (ATMPs), these therapies undergo a centralised, single European Union authorisation by the European Medicines Agency, but the risks, and potential harm to the environment and population at large are weighted in each application, and different interpretations at national level exist. A streamlined procedure is now in place to facilitate a consistent approach for the assessment of the environmental risks of medicines containing genetically modified organisms. This article provides an overview of basic requirements across the EU, an overview of the new streamlined process and discusses available guidance for developers. All these initiatives are aimed to remove hurdles for ATMP developers and facilitate faster access to patients.

Reinforcing Collaboration and Harmonization to Unlock the Potentials of Advanced Therapy Medical Products: Future Efforts Are Awaited From Manufacturers and Decision-Makers

“…The requirements of conducting environmental risk assessment (ERA) are mandatory for medicines containing genetically modified organisms (GMOs), aiming to examine their potentially harmful effects on the ecosystem and human health ( 62 ). Gene therapy vectors, genome editing therapy, and somatic cell therapies whose genetic material has been manipulated will normally be classified as GMOs, while the judgments are made on a case-by-case basis and could vary depending on the interpretations around the environmental and biosafety aspects of GMO-based medicines in different countries ( 62 , 63 ). At the EU level, although the ERA submission for market authorization application is centralized and reviewed by EMA, the REA submission for clinical trials application is processed by national health authorities in each Member State.…”

Advanced therapies are ready to take centre stage: Academia's involvement with regulation needs to raise its game

“…3,9 Although the problem is recognised, the application of the rules to deal with GMOs containing medicines varies widely across Europe. 10 Harmonisation efforts are ongoing, but it may take some time before we approach the full harmonisation that industry and academia are calling for. A radically different approach to solve this problem has arrived with the emergency measures taken by the EC for the COVID-19 pandemic, providing an exception for vaccines containing GMOs, 11 but the derogation applies only to operations necessary to conduct the clinical trial phase and for compassionate or emergency use in the context of COVID-19.…”

“…The unequal treatment of genetically modified organisms (GMO) in members states in the EU has been identified as one of the potential culprits of the slow growth of clinical trials for advanced therapies in Europe 3,9 . Although the problem is recognised, the application of the rules to deal with GMOs containing medicines varies widely across Europe 10 . Harmonisation efforts are ongoing, but it may take some time before we approach the full harmonisation that industry and academia are calling for.…”

Challenging times: Delivering gene therapies and an opportunity for shared learning

“…The EMA drug approval process for gene therapies includes an environmental risk assessment because they are GMOs. 7 As the first Zolgensma ® treatment was administered before the drug had EMA approval, the medicines regulator in Ireland, the Health Products Regulatory Agency, was consulted for advice. Importantly, the project also required the involvement of our Environmental Protection Agency (EPA) for the first dose.…”