Soliris: how was the first human dose set?

The public FDA and EMA reviews of Soliris 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.

Use pharmacology data (target binding, receptor occupancy, in vitro human cells, PK/PD modelling) to estimate the lowest dose expected to have any biological effect (MABEL) and the active dose (PAD), and, for healthy volunteers, start at an exposure below the PAD unless robustly justified.

NOAEL / key findings: Surrogate 26-week mouse study: no treatment effects on any toxicity parameter at either dose; no target organs (EMA assessment report, p. 9)

Full method, every step sourced →