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The three non-commercial developers of ATMPs will benefit from enhanced support from the agency.
The European Medicines Agency (EMA) announced on Feb. 8, 2024 that it has accepted three academic and non-profit organizations into its pilot scheme for advanced therapy medicinal products (ATMPs) development. The pilot’s goal is to guide non-commercial developers of ATMPs through the regulatory and scientific requirements and advance the development of their products to reach the market authorization stage.
The Hospital Clínic de Barcelona was the first participant selected at the start of the pilot. It is developing a medicine to treat patients older than 25 years with relapsed/refractory acute lymphoblastic leukemia. ARI-0001, a chimeric antigen receptor (CAR) product which is based on patients’ own T-cells, was granted Priority Medicine (PRIME) eligibility in December 2021.
In December 2023, EMA selected two new participants from 11 candidates. Berlin Center for Advanced Therapies (BeCAT)–Charité is developing a gene therapy, TregTacRes, based on modified T-cells as an add-on therapy after transplantation.Fondazione Telethon is developing a treatment for 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 authorization applications, and pre-authorization inspections,” the agency stated in a press release.
The agency expects to add an additional two developers to the pilot by the end of 2024. A report detailing the pilot’s initial results is expected in 2025 and will provide areas for improvement in support for academic developers.
Developers wishing to apply for the pilot scheme can contact the agency at firstname.lastname@example.org or complete a form on the agency’s website.