PROTALIX BIOTHERAPEUTICS, INC. (PLX)

[Spideyboy]

The missing word in the transcript marked as [indiscernible] is 'surrogate', and the following word is 'marker' not 'market'. And he says 'where the two' not 'were the two'.

And in a sense yes they were approved on a 'temporary' basis in the sense that both Fabryzyme and Galafold were approved based on surrogate markers and thus while given the temporary approval under the accelerated pathway, the FDA required post-market Phase IV studies to continue in terms of viewing their safety and efficacy after patients started getting the therapies. One would thus assume therefore that given the approval of Fabryzyme in 2003 then one could say the FDA would have been satisfied enough. Galafold while approved in the USA in August will need to do more trials to make the FDA fully happy.
https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm616598.htm
"The efficacy of Galafold was demonstrated in a six-month, placebo-controlled clinical trial in 45 adults with Fabry disease. In this trial, patients treated with Galafold over six months had a greater reduction in globotriaosylceramide (GL-3) in blood vessels of the kidneys (as measured in kidney biopsy samples) as compared to patients on placebo."
" A further study is required to verify and describe the clinical benefits of Galafold, and the sponsor will be conducting a confirmatory clinical trial of Galafold in adults with Fabry disease."

Therefore again you see that these two products were approved by the FDA based on surrogate markers, not actual Kidney function decline like PRX-102. Therefore PRX-102's endpoint of eGFR is the most clinically meaningful end-point to choose and where Bright showed great promise.