so what? Again, this drug has market of 10,000 cases WORLDWIDE, and assuming 70% of drug market shall be in China, India, and Africa - we come to only some 3000 cases in US and Europe. So potential market for this drug is.... 3000*500=$1.5M in sales at best. Great return on investment indeed!!!
"So, where the study could go wrong, obviously you could have some variability in the biopsies and where the samples are taken and how they are read. However, when we have been through this with lots of folks with ourselves a lot as well, but we’ve controlled for that as best we can."
There should not have been placebo effect. It was due to the reading the biopsies so the next thing you should look at is the secondary endpoint where its cut & dry. "absolute percent change in kidney interstitial capillary GL-3 from baseline to month 6 was performed. Taken alone this analysis showed a median reduction of 41% in the migalastat HCl group versus a median reduction of 6% in the placebo group (p=0.093)."
I'm expecting the 12 month data to support approval. In addition, we have Pompe data later this month so the stock will recover in short order.