Advanced-therapy medicinal products

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Advanced-therapy medicinal products (ATMPs) are medicines for human use that are based on gene therapy, somatic-cell therapy or tissue engineering. They offer groundbreaking new opportunities for the treatment of disease and injury.

Unlike converntional medicines, advanced therapies are not made from chemicals or proteins. Asvanced-therapy medicines 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 so that they can be used for a function that is different to its ofiginal essential function 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 advanced-therapy medicines: 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.

For detailed definitions of the different groups of advanced-therapy medicinal products, refer to Regulation (EC) No 1394/2007 and Directive 2001/83/EC

Role of the European Medicines Agency

All advanced-therapy medicines are authorised centrally via the European Medicines Agency. 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.

For more information see Questions and answers.

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 a recommendation on whether or not the European Commission should grant or refuse marketing authorisation of the medicinal product. 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;
  • gives recommendation on 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.

 

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