ATMP: advanced therapy medicinal product
An advanced therapy medicinal product (ATMP) is a gene therapy, somatic cell therapy or tissue-engineered medicine under Regulation (EC) No 1394/2007.
Regulation (EC) No 1394/2007 defines ATMPs by reference to Annex I, Part IV of Directive 2001/83/EC: gene therapy medicinal products, somatic cell therapy medicinal products, tissue engineered products and combined ATMPs that incorporate a medical device. They fall within the mandatory scope of the centralised procedure, so every ATMP marketing authorisation is granted by the European Commission after a CHMP opinion. The Committee for Advanced Therapies (CAT), created by the Regulation, prepares the draft opinion on each application and the CHMP adopts the final one.
The Regulation adds instruments specific to these products. The CAT gives scientific recommendations on whether a product is an ATMP under Article 17, which settles the classification before development goes further. Small and medium-sized enterprises can ask for certification of the quality and non-clinical data of their product under Article 18. Risk management, traceability of the product and its starting materials for 30 years, and follow-up of efficacy and adverse reactions are all stricter than for conventional medicines, and the Commission's guidelines on good manufacturing practice specific to ATMPs apply in Part IV of EudraLex Volume 4. The Regulation also requires a specific post-authorisation follow-up plan for efficacy and adverse reactions, agreed as part of the risk management plan.
Article 28 of the Regulation inserted the hospital exemption into Article 3(7) of Directive 2001/83/EC: an ATMP prepared on a non-routine basis to specific quality standards and used in a hospital in the same Member State under the exclusive professional responsibility of a practitioner for an individual patient falls outside the marketing authorisation requirement, under national authorisation and pharmacovigilance rules. Member States apply the exemption differently, which has created uneven access across the Union.
What goes wrong: starting material and donor documentation that cannot meet traceability requirements; comparability after a manufacturing change during development, which is harder for a cell product than for a small molecule; clinical packages built on single-arm studies without agreed historical controls; and post-authorisation registries that are promised in the RMP but never enrol enough patients. Many ATMPs are also authorised conditionally or under exceptional circumstances, so the obligations continue for years after grant.
Timing
A classification recommendation from the CAT is issued within 60 days of a valid request. The marketing authorisation assessment follows the centralised timetable of 210 active days, with the CAT draft opinion preceding the CHMP opinion, and accelerated assessment of 150 days is available where the criteria are met. Traceability records must be kept for 30 years after the expiry date of the product.
Used in
ATMP holders turn traceability, follow-up and RMP commitments into tracked actions in the obligation-to-action workflow.
Related terms
- Centralised procedure: single EU marketing authorisation via EMAThe centralised procedure grants one marketing authorisation valid across the EU and EEA after a single EMA assessment and a Commission decision.
- Conditional MA: conditional marketing authorisationA conditional marketing authorisation is a one-year, renewable EU authorisation granted on less complete data for medicines that meet an unmet need.
- Exceptional circumstances: marketing authorisation without comprehensive dataAn authorisation under exceptional circumstances is granted when full efficacy and safety data cannot be obtained, with obligations reviewed yearly.
- RMP: risk management planA risk management plan (RMP) describes a medicine's known and potential risks, how they will be studied and minimised, and forms part of the authorisation.
- GMP certificate: proof of good manufacturing practice complianceA GMP certificate is issued by an EU competent authority after an inspection confirms that a manufacturing site complies with good manufacturing practice.
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