They are not going to get even close to 50% patients. This type of rare disease is different from other disease, patients who are currently on ERT are very unlikely to switch unless there is strong data that support the switch. The monotherapy for US trial included only treatment naive patients or those who had discontinued ERT for at least 6 months. So there is no data on patients who switch from ERT to Amigal. Data from EMA trial compared to ERT/switched to ERT will not be available for two more years.
I am along the line with genisi. At minimum, monotherapy isn't going to be as effective as ERT.
I agree with what jq said (about those already on ERT and unlikely it being better). I am not quite as against the drug. I think it has a very good place in the market for those newly diagnosed particularly late onset who probably had mild symptoms till later in life and may not be thrilled at regular IV infusions. I also believe there are probably quite a lot of those people out there (If I recall correctly I think Amicus thought a larger percentage of those people had a mutation that would respond to their chaperone too). The problem is I think it'll take a lot of effort to reach those people. So while sales could keep growing I can't imagine a scenario where (monotherapy) they get to US sales of $100 million till at least 4-5+ years post approval (if it ever gets there monotherapy).
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