PROTALIX BIOTHERAPEUTICS, INC. (PLX)

[Spideyboy]

Can't be sure about the FDA approval by end of year, as in the earnings call it was mentioned that PLX with Chiesi would discuss and seek to submit the paperwork for the Accelerated Approval request in the first half of 2019. It would hopefully take the FDA less than 6 months to review that so that we could hit the end of year mark, but this clearly cannot be guaranteed.

But the good thing, is that if the FDA gives accelerated approval for 102, the that would make it approved for FDA and EMA at similar time-points, given the Balance study is marked as 12 months of data for EMA, and 24 months of data for FDA. Hence a positive for Chiesi as well as PLX so the co-operation on the submission.

Regarding Galafold:

I refer back to this link and extracts from it I provided earlier, this FDA article is from Galafold's FDA approval August 2018:
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."

So yes Amicus is required to run its confirmatory trial for full approval. Whether this specific trial has begun yet is hard to tell, as this requirement was only stipulated by the FDA 4 months ago, this trial is probably in preparation stages and from clinicaltrial.org it would seem this hasn't started yet as I haven't seen anything there.

However, we can see that Amicus does already have a pediatric trial going https://clinicaltrials.gov/ct2/show/NCT03500094?term=migalastat&draw=2&rank=1

This pediatric trial which was started in October 2018 (after this FDA Accelerated Approval in adults), does also take into account change in GB3 and eGFR. So looks like they are taking on board the correct end-points. However again we take into account the smaller maximum 20-30% of the mentioned potential Fabry patient market that have amenable mutations, of which they don't seem to be covering. Also from the Amicus Attract study results it seems they've made it very difficult to understand the impact on eGFR. Probably why the FDA is requiring another study that will clarify this.

https://jmg.bmj.com/content/54/4/288


Regards,C.C. Abbott, it seems after the full interim data provision from the Bright study that she has backed off writing nonsense on PLX and let's hope that remains the case. It would quite frankly be impossible for her to state that the clear reversal in eGFR slope and the statistical significance of p=0.015 is not a very positive outcome for these patients. And given the years of nonsense, good riddance. I'm happy to hear different views, but not when it is actively poorly analysing incorrectly chosen data points to suit the perspectives the writer wishes for whatever motivations they may have for it.