German regenerative therapeutics company t2cure claims to be the first to have received a quality certification for one of its stem cell product candidates under the European Medicines Agency’s (EMEA) regulations for advanced therapy medicinal products (ATMPs). The certification relates to t2cure’s lead autologous bone marrow-derived therapy, t2c001, which is in Phase II development for the treatment of cardiovascular diseases.
“This achievement also proves that the regulations set out by the EMEA for advanced therapy products and the expertise brought together by the EMEA’s Committee for Advanced Therapies effectively assist small- and medium-sized companies in developing advanced therapy products,” says Petra Rueck, Ph.D., CEO, commenting on the ATMP quality certification.
ATMPs are classed as stem cell, gene therapy, or engineered tissue-based therapeutics or diagnostics. In Europe such products are now regulated under a dedicated legislative framework set up at the end of 2008. This framework allows small and medium enterprises (SMEs) to voluntarily apply for certification of the quality of its ATMP, t2cure points out. The firm says that although certification is separate and independent to an MAA, it is beneficial because it does follow the scientific and technical requirements to facilitate a future MAA.
Assessment for quality certification is carried out by the EMEA’s multidisciplinary Committee for Advanced Therapeutics. (CAT). CAT also has responsibility for the evaluation of MAAs for ATMPs. The European approval process for such products is carried out via the centralized procedure.
EMEA stresses that as advanced therapies are often developed by small companies or research units, the ATMP legislation also includes incentives to help the continued development of such products. Such incentives include a 65–90% reduction in fees for requesting scientific advice and for hospitals and SMEs a 50% reduction in marketing authorization application costs. The agency can also provide scientific support to help in the design of pharmacovigilance and risk-management systems.
T2cure is focused on the development of bone marrow-derived cells for use in cardiac repair. The firm says five-year efficacy and safety data from a Phase II trial with lead product t2c001 in severe heart attack patients is expected in the near future. The product is also undergoing pilot clinical trials in chronic ischemic heart disease and in nonischemic dilative cardiomyopathy. T2cure is separately evaluating bone marrow-derived progenitor cells in pilot clinical trials for the treatment of peripheral artery occlusive disease.