Omisirge
omidubicel
Species
Mouse
Longest study
12 wk
Genotox
Not conducted Not conducted
Carcinogenicity
Not conducted Not conducted
Repro
Not conducted Not conducted
First dose
Not conducted Not stated publicly
Speciesopen answer page →
Species
Immunodeficient irradiated NOD/SCID mouse only (xenogeneic human cells)
“In vivo pharmacology, pharmacokinetic, and toxicology studies were conducted to evaluate the activity, distribution, and safety of the product in immunocompromised, irradiated mice.”
Why that species
NOD/SCID chosen for human cell engraftment over other strain in pilot; limited relevance to human homing
“Results showed that NOD/SCID mice were preferable over mice based on cell engraftment levels.”
Surrogate molecule
No surrogate; clinical-like human CF + NF cells tested in immunodeficient mice
“Like omidubicel-onlv, this test article consisted of cells that were expanded for with cytokines and NAM”
Study lengthopen answer page →
Longest pivotal study
12 weeks
“Scheduled Sacrifice Time Points Day 1, Week 4, and Week 12 post-transplantation”
Pivotal GLP studies
One GLP single-dose IV toxicity/biodistribution study in irradiated NOD/SCID mice, sacrifices Day 1, Week 4, Week 12
“The following toxicology study was conducted to evaluate the safety of the test article following administration in the NOD/SCID mouse.”
Route in tox
Intravenous (tail vein), single infusion after total body irradiation
“NOD/SCID mice were administered the product via tail vein injection 24 hours after a TBI dose of 300 cGy according to the study groups below.”
Skipped studiesopen answer page →
Genotoxicity
No standard genotox; cytogenetic/karyotype analyses of CF and NF showed no clonal aneuploidy
“The genomic integrity was evaluated using cytogenic and karyotype analysis. Results showed no difference in the occurrence of chromosomal abnormalities between the CF, NF, and control cells and no evidence of clonal aneuploidy.”
Carcinogenicity
Not conducted (not warranted based on known safety profile)
“No carcinogenicity/tumorgenicity studies were conducted with the test article and were not warranted based on the known safety profile.”
Reproductive & developmental
Not conducted (no reproductive organ findings; conditioning regimen already affects fertility)
“Per the applicant, developmental and reproductive toxicology studies were not conducted because there were no test article-related toxicity findings in reproductive organs as assessed by histopathological analyses”
Safety pharmacology
Not conducted
“No safety pharmacology studies with omidubicel-onlv were conducted.”
First doseopen answer page →
First-in-human starting dose
Not stated in public reviews
NOAEL / key findings
No NOAEL stated; no test article-related adverse effects at up to 10x10^6 CF + 5x10^6 NF cells/mouse; deaths attributed to irradiation
“No test article-related adverse effects were observed in body weights, food consumption, clinical pathology, or histopathology assessments.”
Risksopen answer page →
Immune & cytokine safety
Not assessed nonclinically (immunodeficient model); clinically GvHD dose-safety relationship flat
“The dose-safety relationship is essentially flat suggesting that an increase in dose did not result in an increase in adverse events of interest such as aGvHD and cGvHD.”
Biodistribution & persistence
GLP mouse qPCR: human DNA in all tissues through Week 12, highest femur, spleen, lung, liver, kidney
“At Week 12, relatively high quantities of human DNA were found in femur, spleen, lung, liver, and kidney in the high dose group.”
Modality-specific risk
Genomic instability/tumorigenicity of ex vivo expanded HPCs; engraftment efficacy
“This study indicates that the CF manufacturing procedure, including NAM culture, does not appear to generate chromosomal abnormalities in the regions analyzed when compared to unmanipulated UCB.”