INSTITUTION PROFILE / iPS CELLS & REGENERATIVE MEDICINE
EMA
The EU agency that scientifically evaluates regenerative medicines, but does not authorise them
The European Medicines Agency (EMA) is a body of the European Union. It evaluates advanced therapy medicinal products (ATMPs), meaning regenerative medicines, cell therapies and gene therapies including those made from iPS cells, scientifically, through a centralised procedure. The decision to authorise a sale, however, belongs to the European Commission; the EMA issues a recommendation. Because one authorisation covers the whole EU plus three other European Economic Area countries, any company that wants to sell in the EU has to go through it. This profile looks at one question only: how much influence the EMA actually has over iPS cells and regenerative medicine.
- The EMA in 30 seconds
- What kind of body it is: the four types of influence
- What it does for iPS cells and regenerative medicine
- How far its influence reaches (powers and geography)
- Organisations it works with
- Outlook
- Glossary, references and claim-to-source audit
1. The EMA in 30 seconds
The EMA handles the scientific evaluation of regenerative medicines, but it is not the body that authorises them. The EMA's own pages say that under EU law the agency has no power to authorise sales in EU countries, and that the European Commission issues the legally binding decision Sourced. Even so, its influence is large. Gene therapy, cell therapy and tissue-engineered products (ATMPs) must go through the EMA's centralised procedure, and a single authorisation is valid across the whole EU and three further EEA countries Sourced.
2. What kind of body it is: the four types of influence
Unlike the companies in this series, the institutions that appear in coverage of iPS cells and regenerative medicine do not make cells themselves. What separates them is the route by which they influence the industry, and that gives four types.
3. What it does for iPS cells and regenerative medicine
The EMA groups three kinds of medicine, gene therapies, somatic cell therapies and tissue-engineered products, under the name ATMP (advanced therapy medicinal product) and evaluates them through a centralised procedure Sourced. Depending on how heavily they are processed, iPS cell-derived products would most likely count as either somatic cell therapies or tissue-engineered products (our interpretation).
| Role | What it involves | Document behind it |
|---|---|---|
| How an evaluation runs | The CAT drafts an opinion on quality, safety and efficacy, and the CHMP gives its opinion on whether to recommend approval. The European Commission takes the final decision | The EMA's ATMP overview and the CAT page |
| Mandatory centralised procedure | ATMPs fall under the centralised procedure, in which the EMA carries out the whole scientific evaluation. One application leads to an authorisation valid across the EU | The EMA's page on authorisation of medicines |
| Classification and advice | The CAT issues a scientific recommendation on whether a product counts as an ATMP, and takes part in scientific advice. It also certifies quality and non-clinical data for small and medium-sized enterprises | The EMA's legal framework page and the CAT page |
| How stem cells are treated | Stem cells become an ATMP if they undergo substantial manipulation or are used for a different function from the one they originally had. A 2011 reflection paper named manufacturing consistency, tumour formation and rejection as points to assess | The EMA's ATMP overview |
| First stem cell-containing ATMP | In December 2014 it recommended approval of Holoclar (for limbal stem cell deficiency caused by eye burns) | The EMA press release |
| Dealing with unapproved treatments | In March 2025 the CAT and the Heads of Medicines Agencies (HMA), the body of national agency heads, issued a joint statement on the risks of unapproved advanced therapies | The EMA's ATMP overview |
The ATMP Regulation takes out of its scope any ATMP that is prepared in a hospital, under a doctor's responsibility, for an individual prescription, on a non-routine basis. Manufacture under this exception is authorised by the authorities of the member state, and the product must be used in the same country Sourced. The Regulation also says that whether particular cells, such as embryonic stem cells, may be used is for each member state to decide, and that EU-level rules do not interfere with that choice Sourced. So alongside the route through the EMA, where a product is sold as a product, national treatment of these cases remains.
In 2025 the EMA recommended 104 medicines for marketing authorisation. They included Zemcelpro, a stem cell therapy for blood cancer patients who cannot find a donor Sourced.
4. How far its influence reaches (powers and geography)
| Aspect | What the EMA has | What it does not have |
|---|---|---|
| Powers | The scientific evaluation of ATMPs, classification recommendations, scientific advice and public warnings about unapproved advanced therapies | The decision to authorise a sale (the European Commission) and decisions on price and reimbursement |
| Geography | Its decisions take effect in all EU member states plus Iceland, Liechtenstein and Norway. Because the centralised procedure is mandatory for ATMPs, a company selling in the EU or EEA cannot avoid it | Approvals outside the EU and EEA. Matters left to member states, such as the hospital exemption and embryonic stem cells |
| Scale | 1,035 staff (December 2025) and total income of €607.57 million (2025). A network of experts from across the EU supports its committees | Staff numbers or budget for the CAT or the advanced therapy units alone (not given in the references) |
| Research funding | The EMA's mission is evaluation and supervision; the references we used do not mention it distributing research money | Grants for iPS cell research |
The staff and income figures cover the EMA as a whole, including all human and veterinary medicines Sourced. Also, a product the EMA "recommends" does not reach every patient in the EU straight away. Even after the European Commission's decision, patients may not be able to use it until price and reimbursement have been settled in each country. The EMA plays no part in price or reimbursement decisions Sourced.
5. Organisations it works with
The EMA's evaluation of regenerative medicines involves several committees and the authorities of EU countries. This section shows how the work is divided between organisations rather than listing companies.
The organisations involved and their roles
| Organisation | Role in ATMPs | Source |
|---|---|---|
| CAT (Committee for Advanced Therapies) | Drafts the opinion on an ATMP's quality, safety and efficacy, issues classification recommendations and takes part in scientific advice. It meets monthly | The CAT page |
| CHMP (Committee for Medicinal Products for Human Use) | Adopts an opinion on whether to recommend approval, based on the CAT's opinion | The EMA's ATMP overview |
| PRAC (Pharmacovigilance Risk Assessment Committee) | One of the three committees, with the CAT and the CHMP, involved in the evaluation procedure for ATMPs | The EMA's procedural advice page |
| European Commission | Takes the final decision on the basis of the CHMP's opinion. It does so within 67 days of receiving the recommendation | The EMA's page on authorisation of medicines |
| National authorities | Authorise manufacture under the hospital exemption, and are where price and reimbursement are decided (through national and regional health systems) | The ATMP Regulation and the EMA's page on authorisation of medicines |
6. Outlook
| What the evidence suggests | Primary information it rests on | Category |
|---|---|---|
| The EMA's 2025 annual report says a reform of EU pharmaceutical law has reached political agreement and that it will give advanced therapies a clearer path. This article has not verified when it takes effect or what it contains | The Executive Director's foreword to the EMA's 2025 annual report (regulatory sandboxes, platform technologies, a clear path for advanced therapies, and a reference to the EU Biotech Act proposal) | Not yet confirmed |
| If an iPS cell-derived product is submitted in the EU, the CAT will evaluate it, and tumour formation, rejection and manufacturing consistency are likely to be the focus | The EMA's 2011 reflection paper on stem cell-based medicinal products names these three points | Inference |
| When the first iPS cell-derived ATMP will be approved in the EU | The EMA's documents give no outlook on approvals | Not yet confirmed |
7. Glossary
- ATMP
- Advanced therapy medicinal products. Three kinds of medicine: gene therapies, somatic cell therapies and tissue-engineered products.
- CAT
- The Committee for Advanced Therapies. The EMA committee that drafts opinions on ATMPs.
- CHMP
- The Committee for Medicinal Products for Human Use. The EMA committee that gives the opinion on whether to recommend approval.
- Centralised procedure
- A procedure in which a single application leads to a marketing authorisation valid across the EU. It is mandatory for ATMPs.
- Hospital exemption
- An exception for ATMPs prepared in a hospital, under a doctor's responsibility, for an individual prescription. The member state authorises the manufacture.
- EEA
- The European Economic Area: the EU member states plus Iceland, Liechtenstein and Norway.
8. References
- EMA Who we are: its status as a decentralised EU agency, how it is run, and the 2019 move to Amsterdam. https://www.ema.europa.eu/en/about-us/who-we-are
- EMA Authorisation of medicines: the centralised procedure, the European Commission's decision, the 67-day period, validity across the EEA, the mandatory centralised procedure for ATMPs, and the EMA's lack of involvement in price and reimbursement. https://www.ema.europa.eu/en/about-us/what-we-do/authorisation-medicines
- EMA Advanced therapy medicinal products: Overview: the three ATMP categories, the treatment of stem cells, the 2011 reflection paper, the CAT, and the joint EMA/HMA statement of March 2025. https://www.ema.europa.eu/en/human-regulatory-overview/advanced-therapy-medicinal-products-overview
- EMA Legal framework: Advanced therapies: the ATMP Regulation, classification recommendations and certification for small and medium-sized enterprises. https://www.ema.europa.eu/en/human-regulatory-overview/advanced-therapy-medicinal-products-overview/legal-framework-advanced-therapies
- EMA Committee for Advanced Therapies (CAT) page: its role and monthly meetings. https://www.ema.europa.eu/en/committees/committee-advanced-therapies-cat
- EMA Marketing-authorisation procedures for advanced-therapy medicinal products: the roles of the CAT, CHMP and PRAC, and procedural advice. https://www.ema.europa.eu/en/human-regulatory/marketing-authorisation/advanced-therapies/marketing-authorisation-procedures-advanced-therapy-medicinal-products
- European Union (EUR-Lex) Regulation (EC) No 1394/2007 on advanced therapy medicinal products: the hospital exemption, the amendment of Directive 2001/83/EC by Article 28, and the recital stating that member states' choices are not interfered with. https://eur-lex.europa.eu/legal-content/EN/TXT/HTML/?uri=CELEX:32007R1394
- EMA 2025 Annual Report: 1,035 staff in December 2025, total income of €607.57 million, 104 medicines recommended, Zemcelpro, PRIME, references to the reform of pharmaceutical law, and the founding date of 26 January 1995. https://www.ema.europa.eu/en/documents/annual-report/2025-annual-report-european-medicines-agency_en.pdf
- EMA Press release "First stem-cell therapy recommended for approval in EU" (19 December 2014): Holoclar. https://www.ema.europa.eu/en/news/first-stem-cell-therapy-recommended-approval-eu
9. Claim-to-source audit
| Claim in the article | Category | Source |
|---|---|---|
| Status as a decentralised EU agency, and the 2019 move to Amsterdam | Sourced | Reference 1 https://www.ema.europa.eu/en/about-us/who-we-are |
| The centralised procedure, the European Commission issuing the binding decision, the 67-day period, and validity in the EU and EEA (Iceland, Liechtenstein, Norway) | Sourced | Reference 2 https://www.ema.europa.eu/en/about-us/what-we-do/authorisation-medicines |
| The centralised procedure being mandatory for ATMPs, and the EMA not being involved in price and reimbursement | Sourced | Reference 2 https://www.ema.europa.eu/en/about-us/what-we-do/authorisation-medicines |
| The three ATMP categories, the conditions under which stem cells become ATMPs, the points in the 2011 reflection paper, and the EMA/HMA statement of March 2025 | Sourced | Reference 3 https://www.ema.europa.eu/en/human-regulatory-overview/advanced-therapy-medicinal-products-overview |
| The framework set by the ATMP Regulation, the CAT's classification recommendations, and certification for small and medium-sized enterprises | Sourced | Reference 4 https://www.ema.europa.eu/en/human-regulatory-overview/advanced-therapy-medicinal-products-overview/legal-framework-advanced-therapies |
| The CAT's role (drafting opinions) and its monthly meetings | Sourced | Reference 5 https://www.ema.europa.eu/en/committees/committee-advanced-therapies-cat |
| How the work is divided between the CAT, CHMP and PRAC | Sourced | Reference 6 https://www.ema.europa.eu/en/human-regulatory/marketing-authorisation/advanced-therapies/marketing-authorisation-procedures-advanced-therapy-medicinal-products |
| The conditions of the hospital exemption, and manufacturing authorisation by member state authorities | Sourced | Reference 7 https://eur-lex.europa.eu/legal-content/EN/TXT/HTML/?uri=CELEX:32007R1394 |
| The EU not interfering with member states' decisions on using particular cells such as embryonic stem cells | Sourced | Reference 7 https://eur-lex.europa.eu/legal-content/EN/TXT/HTML/?uri=CELEX:32007R1394 |
| Founding on 26 January 1995, 1,035 staff (December 2025), total income of €607.57 million, 104 medicines recommended, and Zemcelpro | Sourced | Reference 8 https://www.ema.europa.eu/en/documents/annual-report/2025-annual-report-european-medicines-agency_en.pdf |
| The reference to the reform of EU pharmaceutical law (political agreement, a clearer path for advanced therapies) | Sourced | Reference 8 https://www.ema.europa.eu/en/documents/annual-report/2025-annual-report-european-medicines-agency_en.pdf |
| Holoclar being recommended in December 2014 as the first stem cell-containing ATMP | Sourced | Reference 9 https://www.ema.europa.eu/en/news/first-stem-cell-therapy-recommended-approval-eu |
| The four types of institution, and the diagram of the authorisation route | Our calculation | Our own classification and diagram (drawn from references 2, 3 and 5) |
| iPS cell-derived products counting as somatic cell therapies or tissue-engineered products depending on the degree of processing | Inference | This article's reading of the ATMP categories and the description of stem cells in reference 3 |
| The focus of evaluation for iPS cell-derived products being tumour formation, rejection and manufacturing consistency | Inference | This article's reading of the points in the 2011 reflection paper described in reference 3 |
| When the EU pharmaceutical law reform takes effect, and its specific measures for advanced therapies | Not yet confirmed | Neither content nor timing is detailed in reference 8; this article does not estimate them |
| When the first iPS cell-derived ATMP will be approved in the EU | Not yet confirmed | Not stated in references 1 to 9; this article does not estimate it |
| Staff numbers or budget for the CAT or the advanced therapy units alone | Not yet confirmed | Not stated in references 1, 5 and 8; this article does not estimate them |
Last updated 1 October 2026 / Troy Technical
All figures come from the EMA's official website and annual report and from EU legislation. Items marked "Inference" are this article's reading of the primary information. This article is not medical advice.