Marketing Authorisation Application file for ATMPs

Marketing Authorisation Applicant: The people or company applying for a marketing authorisation for a medicinal product.

Marketing Authorisation holder: The people or company benefiting from a marketing authorisation (here for ATMPs, submitted through the “Centralised procedure") to distribute and sell a medicinal product in the European Union.

European Medicines Agency: Under the centralised procedure, companies submit to the European Medicines Agency a single marketing authorisation application file to be assessed by the Committee for Medicinal Products for Human Use (CHMP), the European Medicines Agency's medicines scientific assessment committee.

The European Medicines Agency is also responsible for the scientific assessment of Advanced Therapy Medicinal Products (ATMPs) manufactured at the industrial scale and intended to be placed on the European Union market. In this case, the Committee for Advanced Therapies (CAT) prepares a draft opinion on the quality, safety and efficacy of ATMPs for final approval by the CHMP. The CHMP will then issue and send to the European Commission a final recommendation on whether the medicinal product should be authorised or not.

European Commission: Once the EMA’s Committee for Medicinal Products for Human Use has provided its recommendation, the marketing authorisation of an Advanced Therapy Medicinal Product is granted or not by the European Commission.

Marketing Authorisation Application file: Marketing authorisation application requires from the applicant the submission of a file containing documentation and data related to the medicinal product to be authorised. The file must comply with the Common Technical Document (CTD), a set of specifications for the marketing authorisation application dossier. Under the centralised procedure the application is submitted under a standardised electronic format, the electronic Common Technical Document (eCTD), through an online portal (eSubmission managed by EMA). 

Marketing authorisation: The approval to market a medicine in one, several or all European Union Member States. (Source: EMA Glossary of regulatory terms). 

All medicinal products must have been granted a marketing authorisation prior to be placed legally on the market within the European Economic Area. The purpose of the marketing authorisation is to ensure high quality, and safety and efficacy regarding the benefit/risk balance that shall be positive for the medicinal products.

For Advanced Therapy Medicinal products, a single application is submitted to the European Medicines Agency under the centralised procedure. It will be scientifically assessed by the Committee for Medicinal Products for Human Use (CHMP) as well as other relevant Committees, especially the Committee for Advanced Therapies (CAT). The marketing authorisation issued by the European Commission is valid within the European Union Member States, Iceland, Norway and Liechtenstein for a five-years period.

Centralised procedure: The centralised procedure allows the marketing-authorisation holder to market the medicinal product and make it available to patients and healthcare professionals throughout the European Union on the basis of a single marketing authorisation delivered by the European Commission after the scientific assessment of the European Medicines Agency (EMA).

Common Technical Document: A common format for submitting scientific information when applying for marketing authorisations in the European Union, Japan and the United States. (Source: EMA Glossary of regulatory terms)

Electronic common technical document: A common technical document in electronic format. Abbreviated as eCTD. (Source: EMA Glossary of regulatory terms)

Regulation (EC) No 1394/2007 of the European Parliament and of the Council of 13 November 2007 on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004 (Text with EEA relevance), OJ L 324, 10.12.2007, p. 121-137, CELEX number: 32007R1394

• Original text (available in the 24 official languages of the EU)
• Current version with last amendments (available in the 24 official languages of the EU) 
• Document summary (available in the 24 official languages of the EU)

Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the Community code relating to medicinal products for human use, OJ L 311, 28.11.2001, p. 67-128, CELEX number: 32001L0083, as amended.

• Original text (available in the 24 official languages of the EU)
• Current version with last amendments (available in the 24 official languages of the EU)
• Document summary (available in the 24 official languages of the EU)

Regulation (EC) No 726/2004 of the European Parliament and of the Council of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency (Text with EEA relevance), OJ L 136, 30.4.2004, p. 1-33, CELEX number: 32004R0726

• Original text (available in the 24 official languages of the EU)
• Current version with last amendments (available in the 24 official languages of the EU)
• Document summary (available in the 24 official languages of the EU)

Commission Directive 2009/120/EC of 14 September 2009 amending Directive 2001/83/EC of the European Parliament and of the Council on the Community code relating to medicinal products for human use as regards advanced therapy medicinal products (Text with EEA relevance), OJ L 242, 15.9.2009, p. 3-12, CELEX number: 32009L0120

• Original text (available in the 24 official languages of the EU)

European Union documents

Volume 2 of the publications "The rules governing medicinal products in the European Union" contains a list of regulatory guidelines related to procedural and regulatory requirements such as renewal procedures, dossier requirements for Type IA/IB variation notifications, summary of product characteristics (SmPC), package information and classification for the supply, readability of the label and package leaflet requirements.

• Notice to Applicants Volume 2B - Medicinal products for human use - Presentation and format of the dossier Common Technical Document (CTD) 

This Notice to Applicants (NTA) has been prepared by the European Commission in consultation with the competent authorities of the Member States, the European Medicines Agency and interested parties in order to fulfil the Commission’s obligations with respect to article 6 of Regulation (EC) No. 726/2004, and with respect to the Annex I to Directive 2001/83/EC as amended.

Electronic Application Forms: The use of the electronic Application Forms (eAF) is mandatory for all procedures from 1 January 2016. The eAFs must be used for all applications: authorisations, variations and renewals.

• eSubmission: EU Electronic Application Forms (Module 1.2 application, variation and renewal forms)
• Notice to applicants Volume 2B - Questions & Answers (

User guide for the electronic application form: The user guide and regular update for the electronic application form are available on eSubmission website.

Electronic Common technical Document (eCTD)

• EU Module 1 eCTD Specification (region-specific administrative and product information, version 3.04 April 2021)
• Harmonised Technical Guidance for eCTD Submissions in the EU, version 5.0 December 2021
  • This guidance document is intended to assist pharmaceutical companies with the submission of regulatory information in electronic Common Technical Document format (eCTD) to the National Competent Authorities (NCAs) and the European Medicines Agency (EMA).
• Variations in eCTD format Q&A document, February 2015
  • This document uses a question and answer format to give some guidance when submitting variation applications in eCTD format.
• EU Change Control Process for Change Requests in the Entire Area of Electronic Submissions for Human Medicinal Products, version 2.1 May 2011
• Electronic Submission Change Request Q&A Form

Content and requirements of application forms

• Volume 2B - Module 1.2: Administrative information Application form, Revision 15, September 2021
• Application for variation to a Marketing Application, September 2021
• Application form for renewal of a Marketing Authorisation

European Medicines Agency

• ICH: M 4: Common technical document for the registration of pharmaceuticals for human use: Organisation of CTD 
  • This guideline presents the agreed upon common format for the preparation of a well-structured common technical document for applications that will be submitted to regulatory authorities. It primarily addresses the organisation of the information to be presented in registration applications for new pharmaceuticals (including biotechnology-derived products).
• ICH Topic M 4 - Common Technical Document for the Registration of Pharmaceuticals for Human Use Q&A
• ICH M4Q Common technical document for the registration of pharmaceuticals for human use - quality
• ICH M4S Common technical document for the registration of pharmaceuticals for human use - safety
• ICH M4E Common technical document for the registration of pharmaceuticals for human use - efficacy
• EMEA implementation of electronic submissions: statements of intent

International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH)

The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use brings together the regulatory authorities and pharmaceutical industry to discuss scientific and technical aspects of pharmaceuticals and develop ICH guidelines applied by regulatory authorities.

• The CTD Triangle
• Electronic Common Technical Document Specification, v. 3.2.2., July 2008
  • Current version of the eCTD specification to be used for CTD modules 2-5
• Organisation of the CTD, 15 June 2016
• CTD on Efficacy - Revised guideline, 15 June 2016

More marketing authorisation guidance documents on EMA website here.

1. Marketing Authorisation Applications Submitted to the European Medicines Agency by Small and Medium-Sized Enterprises: An Analysis of Major Objections and Their Impact on Outcomes - Amaouche, Nadia, Hélène Casaert Salomé, Olivier Collignon, Mariana Roldao Santos, and Constantinos Ziogas. Drug Discovery Today 23, no 10 (10/2018): 1801‑1805
   1. Scientific abstract
2. The Electronic Common Technical Document - From Design to Submission - Cartwright, Anthony C. International Journal of Pharmaceutical Medicine 20, no 3 (1/06/2006): 149‑158
   1. Scientific abstract
3. Hurdles in Gene Therapy Regulatory Approval: A Retrospective Analysis of European Marketing Authorization Applications - Carvalho, Marta, Ana Paula Martins, and Bruno Sepodes.Drug Discovery Today 24, no 3 (03/2019): 823‑828 
   1. Scientific abstract
4. View of Drug approval process in US, Europe and India and its regulatory requirements: A Review - Chakraborty, Krishnasis, and Kavita Yadav. International Journal of Drug Regulatory Affairs., s. d., (09/2019): 31-39
   1. Scientific abstract
5. International Marketing Applications according to the ICH CTD Format:  Comparison of Structure and Document Requirements - Gelman, Rita Segal. The Quality Assurance Journal 10, no 2 (2006): 111‑120
   1. Scientific abstract
6. An Overview of the Common Technical Document (CTD) Regulatory Dossier - Jordan, Debbie. Medical Writing 23, no 2 (1/06/ 2014): 101‑105  
   1. Scientific abstract
7. Internationalization of Regulatory Requirements - Juillet, Y. Fundamental & Clinical Pharmacology 17, no 1 (2003): 21‑25
   1. Scientific abstract           
8. The Value and Benefits of the International Conference on Harmonisation to Drug Regulatory Authorities: Advancing Harmonization for Better Public Health  - Molzon, Justina, A Giaquinto, L Lindstrom, T Tominaga, M Ward, P Doerr, L Hunt, et L Rago. Clinical Pharmacology & Therapeutics 89, no 4 (2011): 503‑512
   1. Scientific abstract 
9. The Common Technical Document: The Changing Face of the New Drug Application -  Molzon, Justina. Nature Reviews Drug Discovery 2, no 1 (01/2003): 71‑74
   1. Scientific abstract
10. The International Conference on Harmonization Common Technical Document - Molzon, Justina A. Food and Drug Law Journal 60, no 3 (2005): 447‑452
    1. Scientific abstract
11. Preparing the Common Technical Document for Registration of Pharmaceuticals for Human Use (CTD) - Insights and Recommendations - Roth, Robert I.  Drug Information Journal 42, no 2 (03/2008): 149‑159
    1. Scientific abstract
12. Dossier for Marketing Authorization in the European Union- Todić, Maida. Bosnian Journal of Basic Medical Sciences 3, no 1 (03/2003): 56‑60 
    1. Scientific abstract