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Hospital Clínic de Barcelona, Fondazione Telethon, and Berlin Center for Advanced Therapies were selected out of 11 candidates. The agency will provide these organizations with enhanced support.
Launched in 2022, the scheme aims to support basic research developments into medicines to treat patients in the European Economic Area (EEA). The pilot is open to academic sponsors and non-profit organizations specializing in developing ATMPs.
A spokesperson from the agency said EMA will guide the participants through the regulatory process. The applicant will be provided with a dedicated point of contact from the agency and relevant staff members from different departments will be appointed. During the pilot, all participants will be debriefed and prepared for regulatory submissions.
The objectives of the pilot are primarily to increase the level of ATMP developments into late-stage development and ultimately authorized medicines that can make a difference in patients’ lives in Europe by:
Providing enhanced support to selected applicants from the academic sector during the development of promising advanced therapies to ensure these products will meet regulatory standards in terms of quality, safety and efficacy, and if needed during the planning and submission of marketing authorization applications.
Identifying and addressing potential gaps in the existing regulatory tools and guidance available to applicants, with a particular focus on challenges experienced by developers from the academic sector.
“Of the three participants in the pilot, one is developing a chimeric antigen receptor (CAR)-T cell therapy and two are developing other types of gene therapies. These products are still in development,” a spokesperson for EMA told BioProcess Insider.
“The products in development must target an indication in an area where there is an unmet medical need, because this product may offer a major therapeutic advantage over existing treatments, or benefit patients with no or few treatment options. Taking into account the limited number of products and the timeframe associated with the pilot, the selection panel will prioritize candidates for which the agency’s support could be most impactful.”
The first candidate chosen for development is ARI-0001, a chimeric antigen receptor (CAR) product based on patients’ own T-cells from Hospital Clínic de Barcelona. ARI-0001 aims to treat patients older than 25 years of age with relapsed/refractory acute lymphoblastic leukemia, a type of blood cancer.
In the second round of the scheme, the Berlin Center for Advanced Therapies (BeCAT) Charité’s TregTacRes therapy, and Fondazione Telethon’s Telethon 003 (etuvetidigene autotemcel) were selected.
BeCAT’s TregTacRes therapy is based on modified T-cells and can be used as an add-on therapy after transplantation, whereas Fondazione’s Telethon 003 treats Wiskott-Aldrich Syndrome, a rare, life-threatening immunodeficiency.
Furthermore, the three candidates are eligible to receive fee reductions and waivers as stated by the decision of the agency’s executive director.
The Agency plans to select up to 5 candidates in total by the end of 2024. Developers are encouraged to apply until 31 December 2024, and the results are expected to be available in 2025.
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