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Amicus Therapeutics, Inc. Message Board

  • tinhornsr tinhornsr Feb 28, 2013 8:57 PM Flag

    word from FDA

    they keep pushing back the qtrly, waiting for words from FDA? From last presentation, it seemed they are working to get FDA to okay the 0.3 cut as the new protocol

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    • Who cares? Seriously. Fabry is already an orphan disease, but now they are only hoping at best to treat a small subset of that population with a drug that isn't really all that effective. The market is getting smaller, the odds of approval are getting smaller. What's the point? If they try to sell at as a co-administered product, that will only force greater constraints on price because you can't heap an expensive orphan-priced drug on top of an already expensive infusion therapy. Amicus is simply grasping for straws at this point.

      • 2 Replies to investingveteran
      • What a completely clueless analysis - "a small subset of the population with a drug that really isn't all that effective". First off, migalastat as a monotherapy is expected to cover have of the patient population and for those mutations not amenable to monotherapy (or the ~30% that may not respond to monotherapy and have to go to ERT), migalastat will be used in combination with ERT - in other words, milalastat has the potential to be used in the entire population of Fabry patients. Granted, many Fabry patients may never go on ERT, either because they don't have coverage to pay for it or they don't want to deal with the bi-weekly infusions, but the potential Fabry market for migalastat is larger than that for ERT alone. As evidenced by the percentage of patients that opt to stay on migalastat in the extension phases of the study, there is a clear patient need/demand for an oral drug to control this disease and potentially keep people from having to go on ERT. With respect to effectiveness, it is clear from the Phase II & III data that roughly 2/3rds of the sickest patients respond to the drug (i.e. have their GL3 reduced by 50%) - if you are looking at the measurement noise in the patients with baseline GL3 below 0.3 as a sign that the drug doesn't work, again you are clueless - that is like looking at a flu trial and saying patients with a baseline 1 degree fever did as well on placebo as Tylenol, because a chunk of the placebo patients had a 0.5 degree reduction in their fever from baseline - like a 0.5 degree change in fever can be attributed to normal fluctuations within a patient or measurement inaccuracies, a 0.1 change in GL3 has the same issues. Finally, when you say "the market is getting smaller", the exact opposite is true - this disease has been under-diagnosed and under-treated for years - the ability to diagnose the disease with genetic testing and the availability of an oral treatment will expand the Fabry market over the next decade.

      • You miss the point completely. They are making coadministered products that for fabrys immediately will be given with the enzyme. They will probably sell it for teh same price or marginally higher than the current price and capture the market as their product is superior. For Pompes its saving life threatening allergic reactions so the coadministered product will sell and when they come out with their version with the enzyme it will be given subcutanoeously. This is a longterm story for investors not speculators.

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