Phortas at ATMP Scientific Symposium
We are excited to share that Dr. Darius-Jean Namdjou, our Director, Senior Regulatory Lead at Phortas, recently participated in the European Medicines Agency (EMA) Scientific Symposium on Advanced Therapy Medicinal Products (ATMPs) held at EMA premises, in Amsterdam, Netherlands.
This one-day hybrid event, organized by the Committee for Advanced Therapies (CAT) on the occasion of CAT‘s 15 year anniversary, convened representatives from regulatory agencies, regulatory/development consultancies, pharmaceutical companies and associations, patient associations, and academia.
The involvement of the current and all former CAT chairs made this event especially interesting.
Scientific Symposium Objectives
The main objectives aimed to address at this event were:
Looking back and analyzing the first 15 years of CAT’s existence:
An evolving committee which had no products to assess, when it was put in place, but then witnessed the scientific progress towards some of the most exciting product developments to date
One of the very first ATMP approvals, gene therapy Glybera, was controversial, with differing opinions between CAT, Committee of Human Medicinal Products (CHMP), with the approval process also including a CHMP re-examination procedure.
Current status and outlook to the future:
The vast majority of EMA Priority Medicine (PRIME)-designated products are ATMPs
CAT is involved in significant international convergence initiatives for ATMP regulatory frameworks, with US FDA (cluster meetings every 2 month), further ICH members, regulators from Asia and South America, WHO. Further highly relevant is cooperation within the International Regulatory Frameworks for Cell and Gene Therapies (IPRP). IPRP has by now issued several Reflection Papers on ATMPs.
Key Takeaways
A new EMA guidance on quality, non-clinical and clinical requirements for clinical trials in ATMP development is expected to be published by end of 2024
US based companies dominate the ATMP field, with Asia-Pacific (APAC) companies close behind. Europe is in third place with only about 50% of companies when compared to USA
As a key hurdle for clinical trial approvals and thus trial execution for ATMPs in Europe, remaining national differences in submission documentation despite the coming into force of the EU Clinical Trial Regulation (EU CTR), were identified. These differences mainly concern requirements for Genetically Modified Organisms (GMOs), which are so far regulated nationally. To abolish this hurdle, an update to the EU CTR is planned, shifting the GMO assessment from national to a centralized regulatory responsibility. A GMO assessment should further be completely cut for marketing authorization applications, unless a product is first in class.
Design of clinical trials with the goal to fulfil ATMP marketing authorization requirements is often times considered a weak point by CAT, especially in cases where clinical development is sponsored by Small/Medium companies (SMEs) and academia. It is therefore highly advised to use the regulatory interaction tools offered by EMA such as SME Briefing Meetings and formal Scientific Advice to ensure that the clinical trial design will ultimately meet scientific standards as expected by CAT/EMA.
Luigi Maldini, Director of the San Raffaele Telethon Institute for Gene Therapy in Milano, Italy, provided a highly inspiring presentation on medical progress achieved with cutting-edge stem cell therapy, especially for otherwise fatal rare genetic pediatric disorders. Highly modern stem cell therapy is coupling cell therapy with gene editing or lentiviral gene transfer approaches.
Conclusion
Phortas‘ involvement in this symposium highlights our continuous commitment to staying at the forefront of regulatory developments.
We believe in the enormous potential of ATMPs to shape the future of medicine. We look forward to continuing our engagement with stakeholders and experts on ATMPs and are proud to support our clients in regulatory interactions towards EMA, FDA and further regulators in this quickly evolving field.
Stay tuned for more updates on our participation in such pivotal events and our ongoing efforts to enhance regulatory processes through innovative approaches.
Contact us at info@phortas.com to explore how Phortas can support you with ATMP projects.
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