FOLD/amigal phase 2 extension study in Fabry
Amicus released results on safety, tolerability, and kidney GL-3 in uringe, at the ACMG, end of March, 2009. The added data here are on renal function, another secondary endpoint in the phase III trial.
There were no serious adverse events in phase II trials and I think the problem will be efficacy. Key word used in FOLD's PRs is 'responders'. The definition of a 'good responder' was post-hoc, and there was a lot of variation in effect between 'good responders' and the others, due to many different mutations, I assume.
Btw, unlike the FDA, the EMEA wants a head-to-head phase III trial vs. ERT, and it will be difficult for the chaperone to be as effective as ERT, imo.
