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    • #18970
      Price Wooldridge
      Moderator

      Firdapse is now the only commercially available, FDA-approved medication for LEMS in the United States. It’s in my Lambert-Eaton treatment plan, as it is for many others. Catalyst reports that over 700 patients have used Firsdapse. How effective is it? I hear reports all over the map. Subjective responses to the effectiveness of medications are just that, subjective. That makes results from trial data, done rigorously, give a more quantitative analysis. Here’s our recently published story on the efficacy of Firdapse.

      Pooled Trial Analysis Confirms Efficacy, Safety of Firdapse in Adults

    • #18976
      Ginny Boynton
      Participant

      Despite these trials, there is evidence both anecdotally and from patients’ physicians that Firdapse doesn’t work as well for them as Ruzurgi and some patients are even harmed by Firdapse but not by Ruzurgi. Do you have any thoughts about what will/can be done to make sure these patients don’t lose the mobility they have received through Ruzurgi?

      • #18977
        Price Wooldridge
        Moderator

        @gb Hi Ginny. I think this is a discussion for you and your neurologist. I’ve read plenty of accounts of a medication that worked for one person and not another. Or gave that person an adverse reaction, while the next person has no problem at all. It’s the same for amifampridine. Based on medication availability, insurance coverage, and an individuals specific efficacy and tolerability to the medication, adjustments to the treatment plan may be the only course of action. Again, that will be between you and your neurologist to decide what works best for you.

        • #19004
          Ginny Boynton
          Participant

          Thanks Price. My question relates to whether you think Catalyst will try to produce a version without phosphate salt which can be used by those who are harmed by Firdapse? There is no course of action that can be taken with a person’s neurologist which will help these individuals.

          • #19005
            Price Wooldridge
            Moderator

            @gb Ginny, that’s a good question. I’m not aware of any work in that area but will check next week. My only other thought is having 3,4 Dap compounded at a compounding pharmacy off your neurologist’s prescription. I believe in the UK NHS that is how patients get it. I do know there has been difficulty there with supply due to an ingredient shortage. I don’t have details, but I’ve seen posts regarding that.
            I’ll try and have an answer soon re Catalyst.

          • #19030
            Price Wooldridge
            Moderator

            @gb Hi Ginny. I checked with Catalyst this week and they are currently not testing any formulation of amifampridine without phosphate.

            • #19031
              Ginny Boynton
              Participant

              That’s disturbing news regarding Catalyst not currently working on a formulation of amiframpridine without phosphate. Do you think it doesn’t fully understand how deleterious Firdapse is to some people’s health? If it truly cared about its patients, it would be attempting to do something. Do you think we need to reach out to it so they have a better comprehension of the need?

            • #19033
              Price Wooldridge
              Moderator

              @gb Ginny, I communicated the concerns you expressed to me when I went to get an answer to your question. I’ve always found them responsive to my questions. You my wish to provide them your own perspective.
              Do you know why Jacobus Pharmaceuticals did not pursue FDA approval for their 3,4 Dap they were providing patients? When I was first diagnosed, I couldn’t get my neurologist to get the paperwork done so I could get it. I had to rely on Pyridostigmine and IVIG only.

            • #19034
              Ginny Boynton
              Participant

              Jacobus didn’t seek FDA approval at first because it was providing the drug for free and was happy to do so. I’m sorry your neurologist wouldn’t complete the paperwork to get you on 3,4 DAP. I had almost 18 years of a totally free medication with oversight for my LEMS including blood work and EKGs.

              Jacobus actually received Orphan Drug Approval first but didn’t proceed to FDA approval.

              Who should I reach out to at Catalyst to help them understand the problems with efficacy that many LEMS patients have?

            • #19052
              Price Wooldridge
              Moderator

              @gb You can reach Catalyst via mail at
              Catalyst Pharmaceuticals
              355 Alhambra Circle, Suite 801
              Coral Gables, FL. 33134
              That is their corporate office location.

              You can call them at 305-420-3200.

              or you can email at [email protected].

              The CEO and President is Patrick McEnany.

            • #19095
              Bernie
              Participant

              Price, do you know if there is a pathway for existing patients to obtain Ruzurgi after the mandate takes effect?  It would certainly seem counterproductive to take away one medication from people that really need it.  Perhaps this is an FDA question?

              I have only had Ruzurgi, but am worried that if I transition my life will become more difficult. It would be nice if there were a backdoor to return to where I am now.

              Thanx!

            • #19098
              Price Wooldridge
              Moderator

              @bernie that’s a very pertinent question, and I don’t have an answer. Were I you, I’d first reach out to Jacobus and/or your providing pharmacy and ask them that question. They should be the first to know if/when they must stop providing supply.
              I’ve been on Firdapse and I’m doing great with it. At any point, when you know or feel your supply of Ruzurgi may stop, I would work with your neurologist and Catalyst Pathways to make a transition, if that’s what you and them decide is best for your situation. There are reports all over the map on both these versions of amifampridine. Some love one or the other, some have an adverse reaction to one or the other. Bottom line is how do they work for you specifically. Good luck, and comment here, when you get your answers.

    • #19006
      Ginny Boynton
      Participant

      Yes it is done that way in the UK because neurologists there pushed back and said there was no need for the NHS to pay the ungodly price for Firdapse when 3,4 DAP (Ruzurgi) worked just as well. I don’t believe 3,4 DAP will be permitted to be compounded in the  US. When Jacobus was first working with the FDA 30 years ago there was actually a lengthy meeting about that very subject. If you can get any information about a non phosphate salt version that would be fantastic. I can only take 80 mg. of Firdapse without having to pay even more exorbitant amounts and hope any additional trials will be up to the 100 mg. Ruzurgi provides which is necessary for many patients.

    • #19026
      Gary Chudd
      Participant

      <b>I was on DAP 3,4 for 3 years which I had it compounded at a pharmacy close to where I lived for $140 per month. I was then able to get on a trial study through CU medical in Denver for about 3 years with the same DAP3,4 until January of 2019 when the study wrapped up abruptly and was told a new drug Firdapse which I had to fight the insurance  company with the help of my neurologist and Tafcare which is a fund for aiding in very high priced drugs. I have been trying to get back on DAP 3,4 which is now Ruzurgi which worked better than Firdapse but it is only approved for people 6 to 17 which makes no since the study I was on had no one that young. Any suggestions to get Ruzurgi.</b>

      • #19027
        Price Wooldridge
        Moderator

        @gary-chudd Hi Gary, thanks for your questions. Firdapse, is the only FDA approved amifampridine medication for adults with LEMS in the U.S. It has been previously available as 3,4 Dap compounded, or marketed as Ruzurgi in the U.S. Here, it was prescribed “off-label” for adults as it is only FDA approved for children up to age 17 by the FDA.
        The Orphan Drug Act under which Firdapse was tested and approved allows for 7 years exclusive sales. This encourages all pharmaceutical companies to bear the high cost of testing and development for rare disorders with low patient populations.
        To answer your questions, compounded 3,4 Dap is now on the FDA “do not compound list”. Checking with Catalyst this week, there is no testing underway to provide any formulation other than their brand name, Firdapse. If you’ve had an adverse reaction to Firdapse and can’t take it, the advice is to see your neurologist for other treatment options, or, with your neurologist’s guidance, consider starting a very low dose titration schedule to test your tolerance and adaptability.
        And just as a point of reference, since you mentioned cost, Catalyst has their Pathways Program to help navigate insurance and co-pays. I’ve taken advantage of that help and Firsdapse can be had at little to no co-pays in most cases I’m told.
        I hope this helps.

      • #19032
        Ginny Boynton
        Participant

        Gary,

        I’m sorry you haven’t done well on Firdapse. We have a number of people in our Facebook group who don’t either. My problem is that I take 90 mg. of  Ruzurgi but am only able to take 80 of Firdapse (insurance won’t pay for more. ) We have LEMS patients who have had to be hospitalized on Firdapse but do well on Ruzurgi.

        It’s distressing to hear that Catalyst told Price it wasn’t currently working on a medication that would mimic Ruzurgi and have no phosphate salt.

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