Amicus Therapeutics (NASDAQ:FOLD)
Shares of Amicus were up 30% on Thursday March 19, 2015 after the company stated that it had positive meetings with the FDA and EMA for its migalastat drug in treating Fabry disease. Fabry disease is an inherited disorder caused by a deficiency of an enzyme known as a-galactosidase A — a-Gal A which is responsible for degrading lipids in the body. This enzyme also causes a problem with an accumulation of globotriaosylceramide — GL3. When this acumulation of GL3 occurs it causes the very many symptoms that are associated with Fabry Disease. Such symptoms associated with Fabry disease are heart problems, pain, kidney failure. The disease is a rare disease so it has already received orphan drug designation from regulatory authorities in the U.S., Japan, and European Union.
In Europe the application for approval for a drug is known as receiving a marketing authorization application — MMA . Although The European Union assigns what’s called Rapporteurs who collectively analyze data and help companies with their next steps in the application for approval. This meeting in Europe with the Rapporteurs went so well that Amicus will be able to file its MMA earlier than expected by Q2 2015 instead of having to wait till the 2nd half of 2015.
The FDA on the other hand, here in the U.S., is a little more strict with its approval process. It really wants to approve migalastat because it treats Fabry disease which is an unmet medical need but it will still require that the company provide surrogate endpoints for approval. Surrogate endpoints are clinical endpoints established by the FDA for early efficacy measures to determine whether a drug should be approved early or not. In addition Amicus must satisfy the FDA with a confirmatory phase 4 clinical trial in order to market migalastat. The good news though is that the company can run this confirmatory phase 4 trial after approval so it doesn’t have to rush th
Good move! Heading higher after hours with some decent trades @ 11.08. Should rebound nicely from here with a few tricks to be expected by the Market makers.
#1 pick for perceptive fund. I just bought in today too. Long term hol... when perceptive sells / if there's another huge spike, I'll sell.
Enjoy the ride people.
Thought they would have to retest and take out the stops. Had to be patient to get this near low of the day. Good luck!
Now that MMs have cleared all stops and created a short trap, the stock should start to reverse the trend of the past 2 days and make the next leg up to $18 in the next 2 weeks.
The people who shorted the stock will provide the fuel for the next leg up.
GlaxoSmithKline (NYSE: GSK) will likely be kicking itself for returning its co-commercialization rights to the drug back to Amicus last year, and for selling its equity stake in Amicus this past October. Sanofi's Fabrazyme, an ultra-pricey twice-weekly injection that costs patients around $200,000 per year. Who will make a run at this company first.
I hope your 'sell' is temporary b/c this is a baker brothers conviction stock. They put $ into their positions unlike analysts.
Was just being fair...you were quite clear...
I do not really agree with you though....which is a whole different debate of course...
I'm long on this stock and my DD has me thinking their technology and/or attractiveness as a potential acquisition will indeed move the PPS much higher in the next 2-6 months.
We will at least see some more nice little pops here and there as more data is released and updates come through on the regulatory process.
Not even going to try and market time it....just sitting back and stomaching the swings....
All these price targets have some level of discount applied to them for the risk of regulatory rejection/delay, so upon approval of Migalistat, FOLD is a $20+ stock. From there, their ERT formulations for Fabry and Pompe will double the value over the next 2-3 years. IOW, the ride has just begun - hang on for greater reward.