Spherox: which tox species, and why?

“Comparative analyses of chondrocytes from different species showed that only a limited number of animals including Göttinger mini-pig and Merino sheep were capable of producing stable spheroids.”

“These species were then selected for proof-of-concept studies.”

“In these studies, either human Spheroxs or autologous spheroids produced analogously to human Spheroxs were used.”

“No conventionally designed toxicity studies on Spherox have been performed; given that the product is autologous and that manufacturing takes place without any growth factors or other external stimuli that requires safety testing, such studies are not considered necessary.”

“Reproductive and developmental toxicity studies were not conducted. In line with guidelines on cell-based medicinal products (EMEA/CHMP/410869/2006), the applicant has provided an acceptable justification for omitting these studies: Spherox contains autologous cells with a local mechanism of action, and the risk for biodistribution of cells is limited.”

“including the 6 months GLP study in immunodeficient mice which support the absence of the tumourigenic potential.”

“In these studies, either human Spheroxs or autologous spheroids produced analogously to human Spheroxs were used.”