“Standard in vivo single-dose or repeat-dose toxicity studies were not conducted with afamitresgene autoleucel due to the lack of appropriate animal models for testing of human TCR engineered T-cells.”
✓ VerifiedFDA multi-discipline review, p. 39Source ↗
Pivotal GLP studies
No in vivo tox; in vitro X-scan/MAGE-family peptide cross-reactivity, alloreactivity, normal/iPSC cell co-culture; murine xenograft efficacy
“A comprehensive analysis of cross-reactivity was performed using a peptide library screen with single amino-acid substitutions at each position of the antigenic peptide sequence (X-scan) and peptides from MAGE family member proteins.”
✓ VerifiedFDA summary basis for approval, p. 13Source ↗