New Projects on EMA’s Pilot for Academic and Non-profit ATMPs

In September 2022, the European Medicines Agency (EMA) launched a pilot program designed to support the transition of basic research into medicines that could significantly impact patients’ lives. This initiative was specifically targeted at academic sponsors and non-profit organizations developing Advanced Therapy Medicinal Products (ATMPs).

Throughout the pilot, the EMA committed to providing enhanced regulatory support for up to five selected ATMPs that address unmet medical needs. This includes guiding participants through the regulatory process with the goal of optimizing the development of ATMPs, from best practice principles for manufacturing to planning clinical development that meets regulatory standards.

Recently, the EMA provided an update on the pilot, announcing that three academic and non-profit organizations developing ATMPs have been accepted into the pilot. They are also seeking to include two more developers by the end of the year.

Current Pilot Participants Developing ATMPs

The first participant selected at the pilot’s inception is the Hospital Clínic de Barcelona, developing a Chimeric Antigen Receptor (CAR) product based on patients’ own T-cells for treating relapsed/refractory acute lymphoblastic leukemia. This project has also been granted eligibility for the PRIME scheme to support the development of medicines targeting an unmet medical need.

In the second round, from a total of 11 candidates, the EMA selected two new participants, both developing gene therapy products. The Berlin Center for Advanced Therapies (BeCAT) – Charité is working on a medicine based on modified T-cells, intended as an add-on therapy after transplantation. Fondazione Telethon is developing a therapy based on hematopoietic stem cells for treating Wiskott-Aldrich Syndrome, a rare and life-threatening immunodeficiency.

Benefits of the Pilot Program

Financially, participants in the pilot will benefit from fee incentives for scientific advice, marketing authorization applications, and pre-authorization inspections. From a regulatory development perspective, the EMA will:

– Appoint a dedicated EMA contact point and support team to provide regulatory and scientific support.

– Organize regular teleconferences with the product developers to check progress and identify needs for additional support.

– Optimize the use of existing tools such as pre-submission meetings for supporting regulatory interactions.

– Discuss development plans with pre-planned time and action points for regular assessment.

– Offer support akin to that available to SME developers.

– Assist in proactively identifying questions for which scientific advice should be sought.

How to Apply

Potential developer candidates can apply by completing the application form available on the EMA website. This form includes information on the applicant, type of ATMP, stage of development, product information, justification for addressing an unmet medical need, and an overview of the development plan.

Applications can be submitted via ATMPpilot@ema.europe.eu. An initial pre-selection will be based on the information provided at the time of application, and successful candidates will then be invited to a meeting organized by the EMA Innovation Task Force (ITF) team.

The goal is to select up to five developers by the end of 2024. Applicants should apply by the end of April 2024, as the next pre-selection of candidates is scheduled for this summer. Initial results of the pilot are expected in 2025, after which a report will be published detailing the lessons learned from this initiative and areas for improvement in supporting academic developers.

For more information on the pilot for academic and non-profit developers of advanced therapies, check the official report from EMA here! [https://www.ema.europa.eu/en/news/progress-update-pilot-academic-non-profit-developers-advanced-therapy-medicines]

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