Nonclinical precedent Request an invite
Atlas/Cell therapy/Breyanzi/Safety margin

Breyanzi: what safety margin did regulators accept?

lisocabtagene maraleucel · CD19 CAR-T
Check my plan vs. these
Safety margin at NOAEL
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.
NOAEL / key findings
No NOAEL; long-term safety in animals limited; tissue cross-reactivity showed off-target recognition of low concern
“Animal studies to assess the long-term safety of JCAR017 were limited because the JCAR017 does not survive in immunocompetent rodents and induces graft-versus-host disease in immunocompromised mice.”
✓ VerifiedFDA pharmacology/toxicology review, p. 2Source ↗

Same question, other cell therapies

Encelto

revakinagene taroretcel · CNTF (encapsulated RPE cell implant)
rhCNTF DART NOAEL margins of exposure above 100 vs modelled maximum blood rhCNTF from intraocular implant

Spherox

spheroids of autologous matrix-associated chondrocytes · Autologous chondrocyte spheroids
Minipig study up to 3.2-fold the maximum clinical dose; other studies at doses similar to the clinical dose

Zalmoxis

allogeneic T cells modified with HSV-TK / dLNGFR (nalotimagene carmaleucel) · HSV-TK suicide-gene T cells
Mouse target dose ~two log above proposed clinical dose; this dose caused 100% GvHD mortality (no NOAEL)

Zemcelpro

dorocubicel · UM171-expanded cord blood CD34+
UM171: no toxicity in rat/monkey at doses 8,000-20,000x estimated human exposure

Abecma

idecabtagene vicleucel · BCMA CAR-T
Not stated in public reviews

Alofisel

darvadstrocel · Allogeneic adipose MSC
Not stated in public reviews

Amtagvi

lifileucel · TIL therapy
Not stated in public reviews

Aucatzyl

obecabtagene autoleucel · CD19 CAR-T
Not stated in public reviews

Carvykti

ciltacabtagene autoleucel · BCMA CAR-T
Not stated in public reviews

ChondroCelect

characterised viable autologous cartilage cells · Autologous chondrocytes
Not stated in public reviews

Ebvallo

tabelecleucel · EBV-specific allogeneic T cells
Not stated in public reviews

Gintuit

allogeneic cultured keratinocytes and fibroblasts in bovine collagen · Allogeneic keratinocytes + fibroblasts
Not stated in public reviews

Holoclar

autologous corneal epithelial cells containing limbal stem cells · Autologous limbal stem cells
Not stated in public reviews

Kymriah

tisagenlecleucel · CD19 CAR-T
Not stated in public reviews

Lantidra

donislecel · Allogeneic pancreatic islets
Not stated in public reviews

Laviv

azficel-T · Autologous fibroblasts
Not stated in public reviews

MACI

autologous cultured chondrocytes on porcine collagen membrane · Autologous chondrocytes
Not stated in public reviews

Omisirge

omidubicel · Expanded cord blood HPC
Not stated in public reviews

Provenge

sipuleucel-T · PAP-GM-CSF (autologous APC)
Not stated in public reviews

Rethymic

allogeneic processed thymus tissue · Allogeneic thymus tissue
Not stated in public reviews

Ryoncil

remestemcel-L · Allogeneic BM-MSC
Not stated in public reviews

StrataGraft

allogeneic cultured keratinocytes and dermal fibroblasts in murine collagen · Allogeneic keratinocytes + fibroblasts
Not stated in public reviews

Tecartus

brexucabtagene autoleucel · CD19 CAR-T
Not stated in public reviews

Tecelra

afamitresgene autoleucel · MAGE-A4 TCR-T
Not stated in public reviews

Tregzi

allogeneic Treg immunotherapy with HSPC and T cells (Orca-T) · Allogeneic Treg + HSPC + Tcon
Not stated in public reviews

Yescarta

axicabtagene ciloleucel · CD19 CAR-T
Not stated in public reviews

Summary across all 27 cell therapies →