Ebvallo: which tox species, and why?

“Safety pharmacology studies were not conducted due to the species-specific nature of tabelecleucel and the limited applicability of non-human toxicology models.”

“Because SCID mice are unable to generate functional lymphocytes, engraftment and growth of human lymphocytes and several human tumours are possible.”

“Inoculation of SCID mice with EBV+ BLCL results in development of EBV-induced immunoblastic B cell lymphomas with characteristics similar to EBV-PTLD arising in immunocompromised hosts”

“Single and repeat dose toxicity studies were not conducted as due to the species-specific nature of tabelecleucel these studies would yield no additional information to aid in the understanding of its potential toxicity in humans.”

“Due to the highly species-specific nature of the pharmacology associated with tabelecleucel, nonclinical reproductive/developmental toxicity studies were not submitted with this application.”

“conventional toxicology, carcinogenicity, genotoxicity, mutagenicity and reproductive toxicology studies have not been performed with tabelecleucel.”

“The primary goal of this study was to assess in-vivo homing and EBV specificity and efficacy of tabelecleucel in the presence or absence of IL-2.”