ATMP Pilot Program by EMA - updates
Three organizations have been accepted into the ATMP Pilot Program by EMA.
On Feb. 8, 2024, the European Medicines Agency (EMA) announced the acceptance of three academic and non-profit organizations into its pilot scheme for advanced therapy medicinal products (ATMPs) development. The pilot aims to assist non-commercial developers of ATMPs in navigating the regulatory and scientific requirements, facilitating the advancement of their products toward market authorization.
The Hospital Clínic de Barcelona was chosen as the initial participant at the beginning of the pilot program. We are currently working on a medication that aims to help individuals over the age of 25 who are dealing with relapsed/refractory acute lymphoblastic leukemia. In December 2021, ARI-0001, a chimeric antigen receptor (CAR) product that utilizes patients' T-cells, received Priority Medicine (PRIME) eligibility.
In December 2023, EMA chose two new participants from a pool of 11 candidates. The Berlin Center for Advanced Therapies (BeCAT)–Charité is currently working on a gene therapy called TregTacRes. This therapy utilizes modified T-cells and is intended to be used as an additional treatment option following transplantation. Fondazione Telethon is working on a potential treatment for Wiskott-Aldrich Syndrome, a rare and serious immunodeficiency disorder.
Non-profit academic developers play a crucial role in the development of ATMPs. However, it has been observed that dealing with regulatory requirements can be quite challenging. Although the pilot does not introduce any new regulatory tools, EMA is dedicated to assisting participants in utilizing the existing regulatory tools and development support measures. The chosen pilot participants will receive fee reductions and waivers as outlined in the Agency's Executive Director's decision on fee incentives for scientific advice, marketing authorization applications, and pre-authorization inspections," the agency announced in a press release.
The agency plans to hire two more developers for the pilot by the end of 2024. An anticipated report, set to be released in 2025, will present the pilot's preliminary findings. This report will offer valuable insights into areas that can be enhanced to better support academic developers.
Source: EMA
