Advanced Therapy Medicinal Products (ATMPs) are human medicines based on genes, cells, or engineered tissue. The category includes gene therapy medicinal products, somatic cell therapy medicinal products, and tissue-engineered products, as well as combined ATMPs that incorporate a medical device component. As a rule, placing them on the market in the EU is subject to the centralised marketing authorisation procedure.
Categories of ATMPs
A gene therapy medicinal product contains, or consists of, a recombinant nucleic acid active substance; its use is intended to regulate, repair, replace, add, or delete a genetic sequence. Somatic cell therapy medicinal products consist of cells or tissues that have been substantially manipulated or are not intended to be used for the same essential function in the recipient as in the donor. They are used for treatment, prevention, or diagnosis through pharmacological, immunological, or metabolic action.
A tissue-engineered product contains engineered cells or tissues and is intended to regenerate, repair, or replace human tissue. A combined ATMP additionally includes one or more medical devices as an integral part. Classification can be challenging in borderline cases, for example when cell or tissue components interact with medical device functions. The EMA therefore offers an optional classification procedure.
Marketing authorisation and scientific assessment
ATMPs fall within the mandatory scope of the centralised procedure. The EMA conducts the scientific assessment; the Committee for Advanced Therapies (CAT) has specific expertise for these products. Final marketing authorisation is granted on the basis of the EU procedure. Before placing the product on the market, authorisation is therefore required for the specific medicinal product.
The ATMP Regulation supplements the general medicinal products legal framework with definitions and product-specific rules. Development and authorisation require an integrated concept covering quality, non-clinical and clinical data, manufacturing, and follow-up. Because starting materials are often complex or individualised, traceability, process control, and long-term safety are key planning topics.
Classification as an ATMP cannot be determined solely by a marketing term or the origin of biological material. The statutory definitional criteria and the specific intended use are decisive. For cells and tissues, substantial manipulation and the intended function in the recipient are particularly relevant. Early clarification prevents a development programme from being based on incorrect regulatory assumptions, unsuitable quality documentation, or inappropriate clinical evidence.
The CAT advises on and assesses the scientific specificities of advanced therapies. For the marketing authorisation decision, its contributions are incorporated into the central assessment procedure. Even after authorisation, lifecycle management remains demanding: changes to starting material, manufacturing, or analytics can be clinically relevant. This is particularly true where individualised manufacturing steps or few production batches make standard comparability more difficult.
The specific characteristics of an ATMP also shape communication between the sponsor, manufacturing sites, and trial sites. Clinical and quality-related changes must not be assessed separately if they affect the composition or properties of the administered therapy. An integrated development model provides the basis for consistently controlling manufacturing, release, transport, administration, and traceability throughout the entire course of the trial.
This alignment also facilitates the subsequent analysis of clinical results.
This alignment facilitates continuous regulatory and scientific assessment of the product.
Distinction from biotechnology and biosimilars
Biotechnology is a broad umbrella term for processes that use biological systems or organisms. Not every biotechnologically manufactured medicinal product is therefore an ATMP. Decisive are the legally defined categories of gene, cell, and tissue therapy and, where applicable, combination with a medical device. The existing related entry “biotechnology” describes this broader level, not ATMP regulation.
A biosimilar is also not automatically an ATMP. A biosimilar is a biological medicinal product whose similarity to a reference medicinal product is demonstrated. ATMPs are assessed under their own product category and with product-specific evidence requirements. This entry is also the umbrella term for separate entries on the three ATMP categories and the combined ATMP.
Relevance for clinical trials
ATMP trials must closely integrate clinical design, the manufacturing process, and logistics. Changes to the manufacturing process can affect batch comparability and the interpretation of clinical data. In addition to inclusion and exclusion criteria, dose, and endpoints, Chain of Identity, Chain of Custody, transport conditions, traceability, and long-term follow-up must be planned early. For combined ATMPs, interfaces with medical device legislation are added.
Full-service CROs such as Mediconomics support development strategy, protocol development, site management, monitoring, data management, and safety writing. This also includes coordinating complex logistics, documenting quality-relevant interfaces, and aligning clinical, pharmacovigilance, and regulatory requirements.
Frequently Asked Questions (FAQ)
Are all cell therapies ATMPs?
No. Whether a cell therapy qualifies as an ATMP depends on the statutory criteria, in particular the manipulation and the intended essential function of the cells or tissues.
Is ATMP classification binding?
The EMA offers an optional procedure to obtain early clarity in borderline cases. The marketing authorisation itself then follows the applicable regulatory procedure.
Can an ATMP contain a medical device component?
Yes. A combined ATMP contains one or more medical devices as an integral part and is subject to additional specific requirements.
Regulatory References
- Regulation (EC) No 1394/2007, Article 2 – defines ATMPs, tissue-engineered products, and combined ATMPs.
- Regulation (EC) No 1394/2007, Article 17 – governs the ATMP classification procedure.
- EMA, Advanced therapies: marketing authorisation – explains centralised assessment and marketing authorisation.
