Cell and gene therapies from academia: EMA to help 5 projects going after unmet clinical needs
The European Medicines Agency said Thursday that it’s launching a new pilot program to help academic and other nonprofit researchers developing advanced therapy medicinal products, which includes cell and gene therapies.
Academics have proven to be enormously useful in feeding new products, like chimeric antigen receptor (CAR)-T cell therapies, some of which were first developed by Memorial Sloan Kettering Cancer Center and the University of Pennsylvania, and ushered to the market by biopharma companies. Jean Bennett, formerly with the University of Pennsylvania, also saw her research lead to the approval of gene therapy Luxturna, which Roche now owns.
EMA says that for this pilot it will guide five participants through the regulatory process with the aim to optimize the development of the ATMPs, starting from best practice principles for manufacturing to planning clinical development.
“The pilot’s first participant has already been selected,” EMA said, noting that it’s ARI-0001, a CAR-T product based on patients’ own T cells, developed by the Hospital Clínic Barcelona. The product has already received authorization from the Spanish Agency of Medicines and Medical Devices under a “hospital exemption” approval pathway for the treatment of patients aged >25 years with relapsed/refractory acute lymphoblastic leukemia.
In December 2021, ARI-0001 was granted eligibility to PRIME, EMA’s scheme to support the development of medicines that target an unmet medical need.
“The pilot participants will benefit from all the available regulatory flexibilities and development support measures, such as fee reductions and waivers,” EMA added in a statement. “The progress will be closely monitored, and initial results of the pilot are expected to be available in 3-4 years.”