Case Studies

Application for EU advanced therapy medicinal product (ATMP) classification and orphan drug designation (ODD)

published 2 Feb 2021

Project based on:

Preparation and submission of EU ATMP classification and ODD applications for three products.

Company Description:

Small US-based company focused on developing gene therapy products for the treatment of inherited neurological disorders.

Support required/Problem statement:

Preparation and submission of EU ODD classification applications, including all EMA interactions.

Scope/added complexity:

Preparation of EU ATMP classification and ODD applications for gene therapy products in early stages of development. Owing to this, data sources were subject to change as the preparation of the applications progressed.

The products were ultra-orphan, which added complexity to the preparation of the ODD applications.


All support is provided remotely, except for required attendance at agency meetings.

Activities to date/Issues resolved:

  • Planning project timeframes according to EMA published timelines
  • Advising client regarding EMA ATMP and ODD application requirements
  • Preparation of ATMP and ODD application documents for client review
  • Liaison and discussion with RRL subject matter experts
  • Participation in client meetings to discuss the ATMP and ODD applications content
  • Submission of ATMP and ODD application documents to and subsequent liaison with the EMA
  • Use of the EMA’s IRIS platform for the submission of the ODD applications
  • Liaison with the EMA regarding validation issues and the scientific recommendation of the classification for the products adopted by the Committee for Advanced Therapies (CAT), and designation by the Committee for Orphan Medicinal Products (COMP).


Additional Information:

Reporting to: Director, Regulatory Affairs

Duration of contact: since May 2019

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