Abecma
idecabtagene vicleucel · BCMA CAR-TCountry not stated; first-in-human dose-finding study in patients with relapsed/refractory multiple myeloma (CRB-401)
Lantidra: what was done before first-in-human, and what came after?
donislecel · Allogeneic pancreatic islets
Before first-in-human
First-in-human: where
Not stated in public reviews
Not reported in the public reviews. Reviewers summarise selectively, so this usually means it wasn't discussed publicly, not that it wasn't studied.
Repeat-dose tox before first-in-human
Not stated in public reviews
Not reported in the public reviews. Reviewers summarise selectively, so this usually means it wasn't discussed publicly, not that it wasn't studied.
First-in-human starting dose
No nonclinical basis; target 10,000 EIN/kg from prior Edmonton Protocol clinical experience
“In both UIH-001 & UIH-002 studies a target islet dose of 10,000 EIN/kg was proposed based on the findings of previous experience with Edmonton Protocol for allogeneic islet transplantation.”
Longest repeat-dose systemic studies
Longest repeat-dose study and recovery
No repeat-dose study (no toxicology studies conducted; published literature instead)
“No toxicology studies with LANTIDRA were conducted. The applicant provided publications assessing the overall safety of islet allografts following intraportal transplantation in Module 4 of the BLA.”
Pivotal GLP studies
None; publications on intraportal islet allograft safety (e.g., diabetic mouse liver morphology study) cited instead
“The applicant provided publications assessing the overall safety of islet allografts following intraportal transplantation in Module 4 of the BLA.”
DART package
Species: DART
Not conducted
“No DART studies with LANTIDRA were conducted.”
Reproductive & developmental
Not conducted
“No DART studies with LANTIDRA were conducted.”
PPND / ePPND: when
Not conducted: no DART studies
“No DART studies with LANTIDRA were conducted.”
Carcinogenicity
Species: carcinogenicity
Not conducted; publications in diabetic rats with intraportal islet transplants cited
“No carcinogenicity/tumorigenicity studies with LANITDRA were conducted. The applicant cited two publications that examined the potential for hepatic carcinogenicity following intraportal transplantation of allogeneic islets in diabetic rats.”
Carcinogenicity model
Not conducted; literature: low-number intraportal islet grafts in diabetic rats caused liver tumors, high numbers did not; risk judged low
“This combination of islet number, hyperinsulinism, and hyperglycemia resulted in liver tumors. Transplantation of high numbers of islets did not result in hepatic neoplasms.”
Carcinogenicity timing
Not conducted; published rat islet-transplant literature cited instead
“No carcinogenicity/tumorigenicity studies with LANITDRA were conducted. The applicant cited two publications that examined the potential for hepatic carcinogenicity following intraportal transplantation of allogeneic islets in diabetic rats.”
Same question, other cell therapies
Carvykti
ciltacabtagene autoleucel · BCMA CAR-TCountry not stated; first clinical trial in patients with relapsed/refractory multiple myeloma (LEGEND-2)
MACI
autologous cultured chondrocytes on porcine collagen membrane · Autologous chondrocytesOutside the United States: all clinical studies ex-US (countries not named)
Tecelra
afamitresgene autoleucel · MAGE-A4 TCR-TCountry not stated; first-in-human dose-escalation and expansion study in HLA-A*02 patients with MAGE-A4-positive advanced tumors
Yescarta
axicabtagene ciloleucel · CD19 CAR-TCountry not stated; initial dose-finding study NCI 09-C-0082 in patients with advanced B-cell malignancies
Zemcelpro
dorocubicel · UM171-expanded cord blood CD34+Canada: dose de-escalation study (Study 001) at 2 sites in patients needing allogeneic stem cell transplant
Alofisel
darvadstrocel · Allogeneic adipose MSCNot stated in public reviews
Amtagvi
lifileucel · TIL therapyNot stated in public reviews
Aucatzyl
obecabtagene autoleucel · CD19 CAR-TNot stated in public reviews
Breyanzi
lisocabtagene maraleucel · CD19 CAR-TNot stated in public reviews
ChondroCelect
characterised viable autologous cartilage cells · Autologous chondrocytesNot stated in public reviews
Ebvallo
tabelecleucel · EBV-specific allogeneic T cellsNot stated in public reviews
Encelto
revakinagene taroretcel · CNTF (encapsulated RPE cell implant)Not stated in public reviews
Gintuit
allogeneic cultured keratinocytes and fibroblasts in bovine collagen · Allogeneic keratinocytes + fibroblastsNot stated in public reviews
Holoclar
autologous corneal epithelial cells containing limbal stem cells · Autologous limbal stem cellsNot stated in public reviews
Kymriah
tisagenlecleucel · CD19 CAR-TNot stated in public reviews
Laviv
azficel-T · Autologous fibroblastsNot stated in public reviews
Omisirge
omidubicel · Expanded cord blood HPCNot stated in public reviews
Provenge
sipuleucel-T · PAP-GM-CSF (autologous APC)Not stated in public reviews
Rethymic
allogeneic processed thymus tissue · Allogeneic thymus tissueNot stated in public reviews
Ryoncil
remestemcel-L · Allogeneic BM-MSCNot stated in public reviews
Spherox
spheroids of autologous matrix-associated chondrocytes · Autologous chondrocyte spheroidsNot stated in public reviews
StrataGraft
allogeneic cultured keratinocytes and dermal fibroblasts in murine collagen · Allogeneic keratinocytes + fibroblastsNot stated in public reviews
Tecartus
brexucabtagene autoleucel · CD19 CAR-TNot stated in public reviews
Tregzi
allogeneic Treg immunotherapy with HSPC and T cells (Orca-T) · Allogeneic Treg + HSPC + TconNot stated in public reviews
Zalmoxis
allogeneic T cells modified with HSV-TK / dLNGFR (nalotimagene carmaleucel) · HSV-TK suicide-gene T cellsNot stated in public reviews