How do biotech companies take an ATMP or biologic through the EMA marketing authorisation procedure without getting stuck at the CMC or classification stage?
We guide cell, gene and tissue therapies as well as biologics through the EU procedure: from ATMP classification and CMC strategy to Scientific Advice and the centralised marketing authorisation application at the EMA. Advanced Therapy Medicinal Products fall under Regulation (EC) No 1394/2007 and are assigned to the centralised procedure under Regulation (EC) No 726/2004; a national authorisation is not possible. The real stumbling block rarely lies in the clinical data set but in CMC maturity: with short shelf lives and limited release testing, the manufacturing and characterisation strategy determines whether the dossier withstands the assessment cycles.
- Pharma
- Biotech
Overview
What regulatory specifics apply to ATMPs and biologics?
Support from ATMP classification to marketing authorisation application · Regulation (EC) No 1394/2007, EudraLex Volume 4 Part IV, EMA centralised procedure
Last updated: 2026-06-13
ATMPs and biologics are subject to the strictest requirements in medicinal product regulation. Four points where projects most often get stuck:
- The ATMP Regulation (EC) No 1394/2007 defines the product classes gene therapy medicinal product, somatic cell therapy medicinal product and tissue engineered product. The official categorisation is made via the ATMP classification procedure at the EMA's Committee for Advanced Therapies (CAT).
- ATMPs are mandatorily assigned to the centralised authorisation procedure under Regulation (EC) No 726/2004; a purely national authorisation is not possible. The scientific assessment runs through the CAT before the CHMP issues its recommendation.
- The GMP requirements differ substantially from conventional pharmaceutical manufacturing. ATMPs are governed by the dedicated GMP guidelines in EudraLex Volume 4, Part IV; for biologics, GMP Annex 2 is decisive, and for investigational medicinal products in the clinical phase, the GMP requirements for investigational medicinal products apply. The focus is on sterility and end-to-end traceability from starting material to finished product.
- The Hospital Exemption under Regulation (EC) No 1394/2007 permits non-routinely manufactured ATMPs under national authorisation outside the centralised authorisation. It is narrowly defined and regulated differently in each Member State; in Germany, the Paul-Ehrlich-Institut is the competent authority.
Services
How we support you
ATMP classification & demarcation
Preparation and support of the ATMP classification procedure at the Committee for Advanced Therapies (CAT). Deliverable: a reasoned classification request with categorisation as a gene, cell or tissue product and clarification of borderline cases between medicinal product and medical device.
Authorisation strategy & regulatory roadmap
Definition of the authorisation pathway within the centralised procedure under Regulation (EC) No 726/2004, including assessment of conditional marketing authorisation and authorisation under exceptional circumstances. Deliverable: a documented regulatory roadmap with milestones and authority interactions.
CMC strategy for biologics & ATMPs
Development of the CMC strategy (Chemistry, Manufacturing and Controls): active substance characterisation, reference standards, stability strategy and GMP-compliant manufacturing under EudraLex Volume 4 Part IV (ATMPs) or Annex 2 (biologics). Deliverable: CMC concept and dossier contributions for Module 3.
Learn more →Scientific Advice & authority interaction
Preparation and support of Scientific Advice procedures with the EMA and the CAT. Deliverable: briefing package, list of questions to the authority and follow-up on the assessment as a basis for the development programme.
Learn more →Hospital Exemption & academic ATMPs
Advice for university hospitals and academic institutions on the Hospital Exemption scheme under Regulation (EC) No 1394/2007. Deliverable: assessment of eligibility for national authorisation and establishment of the required quality system.
How we work together
What it comes down to
Regulation (EC) No 1394/2007 does not primarily raise the bar on clinical evidence, but on CMC maturity. Three strands have to come together in the right order: the ATMP classification via the Committee for Advanced Therapies clarifies whether the product falls under the ATMP Regulation at all and is therefore subject to the centralised procedure under Regulation (EC) No 726/2004. The CMC strategy in line with the ATMP GMP guidelines in EudraLex Volume 4 Part IV must demonstrate that active substance, characterisation and release are under control despite short shelf life and limited testing windows. And the clinical programme must fit both. Whoever tackles CMC maturity too late loses it as a critical factor, because, unlike with small molecules, the manufacturing process cannot be separated from the effect here.
This is exactly where we come in. Early Scientific Advice with the EMA and CAT makes visible which requirements the authority sets for comparability and release before dossier modules are written and batches are produced. This shifts the effort into the early strategy, where corrections are cheap, instead of into the assessment, where a Major Objection on CMC delays the entire procedure by months.
Our approach
Our approach
Step
Result
Classification & demarcation
Confirmed product class under Regulation (EC) No 1394/2007, clarified demarcation from medical device or conventional medicinal product.
Authorisation strategy
Defined authorisation pathway within the centralised procedure with roadmap and planned authority interactions.
Scientific Advice
Key points on CMC, non-clinical and clinical programme agreed with the EMA and CAT.
CMC & dossier build-up
CMC strategy under EudraLex Volume 4 Part IV or Annex 2 and prepared dossier modules for submission.
Submission & assessment
Submitted centralised application, structured handling of the CAT and CHMP questions.
Common pitfalls
Where projects commonly fail
The CMC strategy is subordinated to clinical development.
For ATMPs with a short shelf life and limited release testing, the manufacturing and characterisation strategy determines authorisability; CMC maturity reached too late generates the most Major Objections during assessment.
ATMP classification is skipped.
Without early categorisation via the Committee for Advanced Therapies, it remains unclear whether the product falls under Regulation (EC) No 1394/2007 and whether the centralised procedure is mandatory or another pathway is an option.
The Hospital Exemption is treated as a permanent substitute for marketing authorisation.
The scheme under Regulation (EC) No 1394/2007 applies only to non-routine manufacturing within the same Member State and does not replace a centralised authorisation; anyone building a scalable product on it is left without market access at the transition to routine care.
Traceability from starting material to finished product is systematised too late.
Regulation (EC) No 1394/2007 and the ATMP GMP guidelines in EudraLex Volume 4 Part IV require end-to-end traceability for cells and tissues; incomplete documentation of the donor material surfaces at the GMP inspection at the latest.
Scientific Advice is skipped to save time.
Particularly for novel therapies without an established precedent, early alignment with the EMA and CAT replaces costly assumptions about study design and comparability with robust authority expectations.
FAQ
Frequently asked questions
Sources
- Regulation (EC) No 1394/2007 (ATMP Regulation), primary text
- Regulation (EC) No 726/2004 (centralised authorisation procedure), primary text
- EudraLex Volume 4, Part IV (GMP for ATMPs), Annex 2 and GMP for investigational medicinal products, primary text
- Writer material: atmp-biologika-zulassung.md (source content, revised)
- https://theentourage.de/regulatory-compliance/zulassung-atmps-biologika/ (existing page content, revised)
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Case Studies
What this looks like in practice
Regulations & standards considered
- Regulation (EC) No 1394/2007 (ATMP Regulation)
- Regulation (EC) No 726/2004 (centralised authorisation procedure)
- Directive 2001/83/EC (Community code for medicinal products for human use)
- EudraLex Volume 4, Part IV (GMP guidelines specific to ATMPs)
- EudraLex Volume 4, GMP Annex 2 (manufacture of biological active substances and medicinal products)
- EudraLex Volume 4, GMP for investigational medicinal products (Annex 13 / guideline under Regulation (EU) No 536/2014)
- EMA Committee for Advanced Therapies (CAT)
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