The multicentre, randomized Phase III trial will assess the very positive outcome of Phase I/II trial TK007 that resulted in an exceptional improvement in survival of patients by promoting rapid and sustained immune reconstitution, and proved safety and efficacy of haplo-HSCT, thus enabling feasibility of transplantation from partially incompatible family donors. In preparing Phase III, MolMed was supported by the EMEA that provided protocol assistance and scientific advice as granted by the orphan drug designation of TK.
AIFA clearance, the very first one in Italy for a Phase III of a cell/gene therapy, requires the completion of analytical characterization of TK components within the treatment of 20% of patients involved in the study, and follows approval of the clinical protocol, obtained in December 2007 by the Ethical Committee of the first clinical centre involved. MolMed also plans to start a Phase I/II trial of TK in the US, managed by the MD Anderson Cancer Center in Houston in 2008.
Marco Dieci, director of quality & regulatory compliance at MolMed, said: “This approval is particularly meaningful also because TK is one of the very few cell/gene therapies in Phase III all over the world, and thus could be among the very first ones to obtain marketing approval.”