Table 1.
Translational specificities for ATMPs.
| Translational aspects | Conventional medicinal product, including biological | ATMP |
|---|---|---|
| Pharmacodynamics | ||
| Proof-of-concept | Obligatory | Essential, if relevant animal |
| Mode-of-action | Obligatory | models are available |
| Off-target | Less defined | |
| Pharmacokinetics | Obligatory | Distribution |
| Dose to humans | Alloscaled | Alloscaling absent |
| SD acute toxicity | Important | |
| SD chronic toxicity | Not always relevant | Often relevant |
| MD chronic toxicity | Essential, GLP | |
| AESI | Very supportive | Often empirical |
AESI, adverse effects of special interest; MD, multiple dose; MoA, Mode of Action; PD, Pharmacodynamics; PK, Pharmacokinetics; PoC, Proof-of-Concept; SD, single dose.