ATMP Blogpost #1 : Adventurous road towards realizing ATMPs' unprecedented opportunities
ATMPs : promising new therapies
Welcome to the first post of the blog series focused on Advanced Therapy Medicinal Products (ATMPs). We will focus on the most important aspects of ATMPs and address several critical issues faced by ATMP developers and manufacturers. ATMPs are among the most promising new therapies offering unprecedented opportunities to replace existing approaches of treating various medical conditions. Moreover, they often target indications with no pre-existing medical solution.
What specifically are ATMPs?
According to the European Medicines Agency (EMA), the European Union (EU) regulatory body, Regulation 1394/2007, ATMPs include gene and cell therapies (GT & CT), but also tissue engineered products (TEP). For those of you who are more familiar with the FDA regulatory environment, the Centre for Biologics Evaluation and Research is the body dealing with tissue, cell and gene therapies in the US. Since ATMPs often employ cells, an important distinction has to be made, depending on the cell source. An allogeneic therapy is one where the cells are obtained from a donor, that is not the recipient of the therapy itself. In case of autologous products, the patient will be treated using their own cells.
Although extremely innovative, ATMPs still suffer from a number of challenges rooted in the complexity of the products themselves, which makes it intrinsically difficult to deal with. The challenges we are focusing on in this series are:
1. Regulations – in the EU a centralized regulatory framework exists, however Good Manufacturing Practice (GMP) rules continue to be a demanding issue to be navigated, despite the existing Part IV GMP guidelines dedicated to ATMPs. Moreover, in some cases genetically modified organism (GMO) regulations of EU member states can prevent the marketing of certain ATMPs.
2. Reimbursement – the price tag of an ATMP can rise to hundreds of thousands of euros per treatment dose, which is totally unaffordable for a single patient. Therefore, alternative payment models for innovative medicines are being developed (most notably are ‘value-based payments’).
3. Manufacturing – the inherent variability and complexity of raw materials (cells) as well as the often-personalized nature of the final product make this extremely challenging for manufacturing departments and regulatory affairs offices across the globe.
4. Clinical studies – some ATMPs target orphan indications, which poses further challenges in regard to clinical trial design. However, EMA offers some solutions to support the marketing of such products under a conditional authorization.
Given the above, it is not surprising that despite the promising opportunities offered by this novel category of medicines (the ATMPs), there are only a few of them available on the European market.
Where and how does Antleron fit in?
We are a team of bright and creative minds that challenges the current paradigms of advanced therapy manufacturing by combining the Quality-by-Design methodology with innovative technologies, such as biomaterials, 3D-printing, soft sensing and bioreactors. Through partnerships and collaborations, we aim to support ATMP manufacturers and developers to accelerate the engineering of advanced therapies, eventually empowering patients with personalized and regenerative care.
We envision to bring our expertise together with all kinds of like-minded parties to develop complex tissues and organs. Not only for research purposes, but also to accelerate personalized medicine from lab to patient.
ATMPs offer huge opportunities to increase treatment efficacy. Companies, research institutions and regulatory bodies are tackling the various remaining hurdles to allow for a more continuous stream of ATMPs entering the market. Stay tuned for the upcoming blogpost on the ATMP regulatory environment: learn which regulations are key in the context of ATMPs and discover the global ATMP regulatory landscape and its implications. Later posts in the blog series will cover cell therapy, reimbursement, tissue-engineered products, manufacturing, gene therapy, etc.