Makale
Cell and gene therapies in the EU – Between scientific excellence and regulatory caution
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Zaklina Buljovcic
Explore how the EU, U.S., and Asia Pacific are shaping pathways for ATMP and cell and gene therapy development, balancing scientific rigor, regulatory speed, and commercialization
The EU as a group of 27 countries has a high scientific and therapeutic standard for innovative medicines including advanced therapy medicinal products (ATMPs). At the same time, while the European Medicines Agency (EMA) decision-making structures are highly science-based, they can also be rigid and conservative, as reflected in recent withdrawals and negative opinions, including for the tissue-engineered product (TEP), Jelrix. Failure in that case was connected to use of a single arm study due to the lack of suitable comparators.1 However, there are tools in place to speed up innovative development like academia briefings2 and Innovation Task Force briefing meetings,3 Scientific Advice meetings, PRIME scheme,4 and others.5
The commission is seeking to gain further speed for innovative therapy development with upcoming adaptations in their new pharma legislation.6 Speeding up and making approaches less complicated is highly important given that regions in other parts of the world are doing so.
The commission is seeking to gain further speed for innovative therapy development with upcoming adaptations in their new pharma legislation.6 Speeding up and making approaches less complicated is highly important given that regions in other parts of the world are doing so.
The U.S. Food and Drug Administration (FDA) has its cell and gene therapy (CGT) program7 and introduced special programs like the regenerative medicine advanced therapy (RMAT) designation,8 Platform Technology Designation Program,9 flexible requirements for CGTs and recently started clinical trial application transformation with real-time clinical trials.10
Asia Pacific is gathering momentum in the cell and gene area. Australia is enabling faster and less complex clinical trial submission and conduct, along with a more feasible and pragmatic pathway to marketing authorization through its risk-based approach for CGTs.11 Frameworks for CGTs are rapidly evolving in China, Singapore and other countries, with some incorporating risk-based product classifications and innovative reimbursement schemes to enable faster commercialization. However, there is some uncertainty over the speed of development in some markets, given potential serious risks.12
Asia Pacific is gathering momentum in the cell and gene area. Australia is enabling faster and less complex clinical trial submission and conduct, along with a more feasible and pragmatic pathway to marketing authorization through its risk-based approach for CGTs.11 Frameworks for CGTs are rapidly evolving in China, Singapore and other countries, with some incorporating risk-based product classifications and innovative reimbursement schemes to enable faster commercialization. However, there is some uncertainty over the speed of development in some markets, given potential serious risks.12
Cell and Gene Therapies in the EU – Strategies to speed up
There will be changes coming with the New Pharma Regulation and New Pharma Directive.13 These include:
- A “One-Stop-Shop" for Borderline Products (BPs) omitting the specific advanced therapy medicinal product (ATMP) scientific recommendation and enabling classifications of all kinds of BPs in early development
- The Committee for Advanced Therapies (CAT) being abolished as a committee and transferred to an expert group, strengthening the Committee for Medicinal Products for Human Use (CHMP) as the only decision maker for medicinal products in the centralized procedure
- The assessment phase for the centralized procedure is being shortened to 180 days
- Adaptations to the Hospital Exemption to allow better control and increase transparency
- Introduction of regulatory sandboxes
Additionally, the upcoming Biotech Act14 is in the making and will further introduce:
- Shortening of authorization timelines for multinational clinical trials
- Using a core Investigational Medicinal Product Dossier (IMPD) across trials
- Enabling risk-based genetically modified organism (GMO) exemptions in trials
- Adaptation of the ATMPs definitions (adding viral vectors and providing more flexibility with delegating acts to adapt the TEP definition) within scope
- Centers of excellence for ATMPs as well as protected testing environments
- Strengthening regulatory sandboxes for development of innovative therapies
- A transparent Union status repository for biotech products. This should compile relevant opinions, recommendations, decisions and guidance, thus fostering transparency, consistency and mutual learning across EU and national authorities.
This will hopefully lead to:
- Faster clinical trial approvals, with risk-based GMO exemptions providing a significant relief to the field
- Simplification and faster central marketing authorization approvals
- Opportunities to discuss borderline cases within regulatory sandboxes, including emerging engineered living materials (ELMs)
- Better exchange between member states and more transparency of published information
However, there are downsides as well. For example, when the CAT ceases to exist, the group of experts will be transferred to a smaller expert group with no decision rights. This might have a negative impact on some products, such as TEPs a usually lower risk group of ATMPs but very complex in terms of characterization, and the result may be that some TEPs won’t be available to European patients. It is possible that the Biotech Act and use of regulatory sandboxes will help to support TEPs in the future.
Faster trial and marketing authorization procedures, which include a less generous use of clock stop extensions, might also lead to increased refusals because biotech companies, smaller ones in particular, might not be able to answer questions in the remaining clock-stop periods. This requires sponsors to better prepare their dossiers and to align with the regulators using scientific advice before submission to the EMA.
Based on our observations, in July 2026, 33 ATMPs have been granted marketing authorization in the EU, but nine of them have been withdrawn. While some withdrawals were driven by unfavorable clinical results or manufacturing facility issues, most have been withdrawn due to commercial reasons. Part of this commercial challenge is connected to health technology assessment (HTA) and reimbursement issues, and whether the Joint Clinical Assessment (JCA) process can improve this remains to be seen.
Looking Ahead
Europe is clearly at a crossroads. Its scientific excellence is unquestioned. The ambition to modernize the regulatory framework is evident. The key question is whether the forthcoming reforms will strike the right balance between robust patient protection and regulatory agility.
For developers of ATMPs, success in Europe will increasingly depend on early strategy, deep regulatory expertise, and proactive engagement with evolving frameworks including the regulatory sandboxes approach. Navigating this transition effectively will be critical to ensure that innovative ATMPs reach European patients.
For developers of ATMPs, success in Europe will increasingly depend on early strategy, deep regulatory expertise, and proactive engagement with evolving frameworks including the regulatory sandboxes approach. Navigating this transition effectively will be critical to ensure that innovative ATMPs reach European patients.
*Kaynaklar aşağıda listelenmiştir
About the author:
Dr. Zaklina Buljovcic, Director, Principal Consultant, at Cencora is a regulatory specialist for innovative therapies, especially advanced therapy medicinal products with 18 years of experience in the field and 22 years in the regulatory business.
Yasal Uyarı:
Bu makalede verilen bilgiler yasal tavsiye niteliğinde değildir. Cencora, Inc., okuyucuları tartışılan konularla ilgili mevcut bilgileri gözden geçirmeye ve bunlarla ilgili kararlar alırken kendi deneyim ve uzmanlıklarına güvenmeye şiddetle teşvik eder.
Sources:
1. EMA. Jelrix. https://www.ema.europa.eu/en/medicines/human/EPAR/jelrix
2. EMA. Academia. https://www.ema.europa.eu/en/partners-networks/academia
3. EMA. Innovation Task Force briefing meetings. https://www.ema.europa.eu/en/human-regulatory-overview/research-development/innovation-task-force-briefing-meetings
4. EMA. PRIME: priority medicines. https://www.ema.europa.eu/en/human-regulatory-overview/research-development/prime-priority-medicines
5. EMA. Scientific advice and protocol assistance. https://www.ema.europa.eu/en/human-regulatory-overview/research-development/scientific-advice-protocol-assistance
6. EMA. Reform of the EU pharmaceutical legislation. https://www.ema.europa.eu/en/about-us/what-we-do/reform-eu-pharmaceutical-legislation
7. FDA. Cellular & Gene Therapy Products. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products
8. FDA. Regenerative Medicine Advanced Therapy Designation. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/regenerative-medicine-advanced-therapy-designation
9. FDA. Platform Technology Designation Program for Drug Development, May 2024. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/platform-technology-designation-program-drug-development
10. FDA. FDA Announces Major Steps to Implement Real-Time Clinical Trials, April 2026. https://www.fda.gov/news-events/press-announcements/fda-announces-major-steps-implement-real-time-clinical-trials
11. TGA. Clinical Trial Notification (CTN) scheme, Updated April 2025. https://www.tga.gov.au/products/unapproved-therapeutic-goods/access-pathways/clinical-trials/clinical-trial-notification-ctn-scheme
12. Science. Second gene-editing death in China disclosed after long delay, July 2026. https://www.science.org/content/article/second-gene-editing-death-china-disclosed-after-long-delay
13. Council of the European Union. Regulation laying down Union procedures for the authorisation and supervision of medicinal products for human use and establishing rules governing the European Medicines Agency, Feb 2026. https://data.consilium.europa.eu/doc/document/ST-6366-2026-INIT/en/pdf
14. European Commission. Proposal for a Regulation to establish measures to strengthen the Union's biotechnology and biomanufacturing sectors, Dec 2025. https://health.ec.europa.eu/publications/proposal-regulation-establish-measures-strengthen-unions-biotechnology-and-biomanufacturing-sectors_en
