Amtagvi (lifileucel): what was done before first-in-human, and what came after?

Precedia/Cell therapy/Amtagvi/Tox package

Amtagvi: what was done before first-in-human, and what came after?

lifileucel · TIL therapy

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

Not stated in public reviews

Why it's blank

The public FDA and EMA reviews of Amtagvi don't say how the first human dose was chosen. That rationale is in the IND, which is confidential, so it is missing for most approved products.

How it's usually set for cell therapies

FDA expects preclinical proof-of-concept and toxicology studies to identify active dose levels and support the choice of starting dose and escalation. The 2013 guidance gives no numeric formula or scaling rule for the starting dose.

“Selection of potential starting dose level, dose-escalation schedule, and dosing regimen for clinical trials.”

✓ VerifiedFDA 2013: Preclinical Assessment of Investigational Cellular and Gene Therapy…, p. 7Source ↗

This product's starting point

NOAEL / key findings: No NOAEL; no toxicology studies; no target-organ findings available (FDA pharmacology/toxicology review, p. 13)

Full method, every step sourced →

Longest repeat-dose systemic studies

Longest repeat-dose study and recovery

No repeat-dose study (no toxicology studies conducted)

“No toxicology studies were conducted for AMTAGVI.”

✓ VerifiedFDA pharmacology/toxicology review, p. 13Source ↗

Pivotal GLP studies

None; five published pharmacology papers (TIL prognosis, expansion, cytotoxicity, murine tumor models) submitted in Module 4

“No nonclinical pharmacology studies were performed for AMTAGVI.”

✓ VerifiedFDA pharmacology/toxicology review, p. 4Source ↗

DART package

Species: DART

Not conducted (product characteristics and biological activity)

“No DART studies were conducted for AMTAGVI. Given the characteristics and biological activity of AMTAGVI, this omission is acceptable.”