Academic and non-profit developers of advanced therapy medicines (ATMP) supported by EMA in a pilot scheme

EMA has accepted three academic and non-profit organisations developing advanced therapy medicinal products (ATMPs) into a pilot scheme, in which they benefit from enhanced support from the Agency.

The aim is to guide non-commercial developers of promising ATMPs addressing unmet medical needs through the regulatory and scientific requirements in the European Union (EU) and better understand their needs to enable them to advance the development of their medicines and eventually reach the Marketing authorisation application

The first participant selected to join at the start of the pilot is the Hospital Clínic de Barcelona which is developing ARI-0001, a chimeric antigen receptor (CAR) product based on patients’ own T-cells. The medicine is intended for the treatment of patients older than 25 years with relapsed/refractory acute lymphoblastic leukaemia, a type of blood cancer. In December 2021, it was granted eligibility to PRIME, EMA’s scheme to support the development of medicines that target an unmet medical need.

In a second round which finished in December 2023 a total of 11 candidates had expressed their interest to join the pilot. EMA selected two new participants, both developing gene therapy medicines.

TregTacRes, a medicine based on modified T-cells, intended for use as add-on therapy after transplantation, is being developed by Berlin Center for Advanced Therapies (BeCAT) – Charité.

Telethon 003 (Etuvetidigene autotemcel) is developed by Fondazione Telethon and intended for the treatment of Wiskott-Aldrich Syndrome, a rare, life-threatening immunodeficiency.

Non-profit academic developers are a major contributor to the development of ATMPs, but experience has shown that navigating regulatory requirements can be challenging. While the pilot does not introduce any new regulatory tools, EMA is committed to support participants in leveraging the use of existing regulatory tools and development support measures.

The selected pilot participants will benefit from fee reductions and waivers as stated in the Decision of the Agency’s Executive Director on fee incentives for scientific advice, marketing authorisation applications and pre-authorisation inspections.

Initial results of the pilot are expected in 2025. Upon completion, a report will be published on the lessons learnt from this initiative and the areas for improvement in support to academic developers.

Many academic ATMPs under development in Europe rely on hospital exemption (HE). However, EMA notes that “there is no direct link between HE and the academic pilot. The aim of the pilot is to increase translation of products reaching patients in the EEA via the conduct of clinical trials and ultimately regulatory approval via an enhanced collaboration with academic developers; this is regardless of whether these products may have been made available under an HE in one or several countries in the EU".

The Directive proposed by the European Commission as part of the revision of the general pharmaceutical legislation includes reforms to increase the regulation of ATMP development under HE, including “measures for collection, reporting of data as well as review of these data yearly by the competent authorities and their publication by the Agency in a repository.

Furthermore, the Agency should provide a report on the implementation of hospital exemption on the basis of contributions from Member States in order to examine whether an adapted framework should be established for certain less complex ATMPs that have been developed and used under the hospital exemption.