Advanced-therapy medicinal products (ATMPs) are medicines for human use that are based on genes or cells. They offer groundbreaking new opportunities for the treatment of disease and injury.
ATMPs can be classified into four main groups:
- gene-therapy medicines: these contain genes that lead to a therapeutic, prophylactic or diagnostic effect. They work by inserting 'recombinant' genes into the body, usually to treat a variety of diseases, including genetic disorders, cancer or long-term diseases. A recombinant gene is a stretch of DNA that is created in the laboratory, bringing together DNA from different sources;
- somatic-cell therapy medicines: these contain cells or tissues that have been manipulated to change their biological characteristics or cells or tissues not intended to be used for the same essential functions in the body. They can be used to cure, diagnose or prevent diseases;
- tissue-engineered medicines: these contain cells or tissues that have been modified so they can be used to repair, regenerate or replace human tissue;
- combined ATMPs: these contain one or more medical devices as an integral part of the medicine. An example of this is cells embedded in a biodegradable matrix or scaffold.
Role of the European Medicines Agency
All advanced-therapy medicines are authorised centrally via the European Medicines Agency (EMA). They benefit from a single evaluation and authorisation procedure.
As in the case of all medicines, the Agency continues to monitor the safety and efficacy of advanced-therapy medicines after they are approved and marketed. The Agency also gives scientific support to companies to help them design pharmacovigilance and risk management systems used to monitor the safety of these medicines.
Committee for Advanced Therapies
The Agency's Committee for Advanced Therapies (CAT) plays a central role in the scientific assessment of advanced-therapy medicines. It provides the expertise that is needed to evaluate advanced-therapy medicines.
During the assessment procedure, the CAT prepares a draft opinion on the quality, safety and efficacy of the advanced-therapy medicine. It sends this to the Committee for Medicinal Products for Human Use (CHMP). Based on the CAT opinion, the CHMP adopts an opinion recommending or not the authorisation of the medicine by the European Commission. The European Commission makes its final decision on the basis of the CHMP opinion.
The CAT also:
- gives recommendations on the classification of advanced-therapy medicines;
- evaluates applications for certification of quality and non-clinical data for SMEs, following which the Agency issues a certificate;
- contributes towards giving scientific advice on advanced-therapy medicines;
- is involved in any procedure regarding the provision of advice for undertakings on the conduct of efficacy follow-up, pharmacovigilance and risk-management systems of ATMPs;
- advises, at the request of the CHMP, on any medicinal product which may require, for the evaluation of its quality, safety or efficacy, expertise in ATMPs;
- assists scientifically in the elaboration of any documents related to the fulfilment of the objectives of Regulation (EC) No 1394/2007;
- contributes towards an environment that encourages the development of advanced-therapy medicines;
- provides, at the request of the European Commission, scientific expertise and advice for any initiatives related to the development of innovative medicines and therapies.
Stem cells are naturally occurring cells in the body that have the ability to divide and produce a range of different cell types. Stem cells are important in the growth and development of the body, as well as in repair after injury. The past few years have seen a dramatic increase in the level of knowledge about stem cells, and research and investment into their uses in medicines.
Stems cells are categorised as ATMPs because these cells undergo substantial manipulation or are used for a different essential function. They can be somatic-cell therapy products or tissue-engineered products, depending on what the medicine is intended to do in the body.
EMA follows research into the use of stem cells in medicines very closely and is responsible for assessing marketing-authorisation applications for medicines containing stem cells.
The Agency has been advising companies developing stem-cell-based medicines for a number of years:
- the CAT has classified a number of medicines containing stem cells as ATMPs;
- the Scientific Advice Working Party has given advice on the development of at least seven medicines containing stem cells;
- the Committee for Orphan Medicinal Products (COMP) has granted orphan designation to a number of medicines containing stem cells for the treatment of rare diseases.
- in December 2014, EMA recommended the first ATMP containing stem cells for approval in the European Union.
In February 2011, the Agency published a reflection paper on stem cell-based medicinal products. The paper stressed the fact that companies developing medicines including stem cells need to pay close attention to the way the medicines are manufactured, to make sure that the final medicine is as consistent and reproducible as possible. Pre-clinical and clinical testing also needs to take account of the cells' properties, ensuring that the possible risks of tumour development and rejection by the body are studied adequately and balanced against their benefits for patients.
The reflection paper was adopted by the CAT on 14 January 2011. This followed a public consultation between March and June 2010 and discussion at a public workshop in May 2010 involving 230 participants from academia, industry and regulatory authorities.
- Legal framework
- Advanced-therapy development and support to companies
- Marketing-authorisation procedures for advanced-therapy medicinal products
- Guidelines relevant for advanced-therapy medicinal products
- Meetings with stakeholders and other organisations
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- Advanced-therapy medicinal products and the CAT (22/05/2013)
- Reflection paper on stem cell-based medicinal products (03/02/2011)