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Diquafosol Info

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  • Diquafosol Info

    Thought I would offer a bit of an update on diquafosol, as I have been following it pretty closely since its mid-phase trials. For those who do not know, this is a drop made by Inspire Pharm, which stimulates the P2Y2 receptors, the proteins on the surface of cells in our lids which make them secrete mucin, lipid, and water.

    Diquafosol has not been rejected by the FDA (or accepted) as yet. Certain elements of the diquafosol Phase III study were positive (though the primary endpoint was not), and because of the high demand for this kind of drug and the favorable safety profile, I think there is a chance (33% maybe?) of it getting approved. The parent company found in one trial that it seems to improve corneal wound healing, and so intends to do a study aiming in that direction, and going for approval for that indication.

    As most of you probably know, if a drug gets approved for ANY indication, it can (and will) be prescribed by doctors for whatever reason they want to prescribe it.

    If you are interested in letting the FDA know your opinion on the subject of whether or not it should be approved (the answer is "YES!!!--even if it works for 1 patient on the board, that would make it worthwhile to me, since the drug has been very clearly proven safe), you can send them a personal note via this page:

    I think a personal note about your own problem and the lack of effective medicines to deal with this problem would be most effective. I don't want to be a shill for any company, and I do not own stock in Inspire, but I believe that the more drugs become available, the better chance you and I have of finding something that helps.

    My 2 cents.

  • #2
    Might be interesting to analyze the data in another way. Even though, on average, dry eye was not improved in the sample, there might well be a cluster of patients who showed significant improvement. What is it about those patients that led to improvement (assuming they exist)? Whatever it is, those characteristics become the basis on which the drug should be prescribed, once it's approved.



    • #3
      Raj, you have hit on it exactly. We have this way of evaluating the success of drugs, which completely ignores the fact that in some cases, a smallish percentage of people (say, 10%), get a dramatic or at least very good response from the drug, while the majority get none. Restasis is a perfect example--did not help me, or many of us on this board, but I know some people for whom it has been a wonder drug, including my own mother.

      We need to get better at statistically analyzing data so that we don't miss the small groups that benefit from a particular drug. If we have 1000 dry-eye drugs on the market, and each one only works on 1% of people, we would still cover > 99.9% of people out there.