• The Firdapse Ruzurgi Conundrum

    Posted by price-wooldridge on October 6, 2021 at 1:21 pm

    With the recent legal judgement in the U.S. regarding Firdapse and Ruzurgi, aka Catalyst Pharmaceuticals and Jacobus Pharmaceuticals, the opportunity for difficulty in our LEMS community is up again. Here is the judgement I refer to, if you’ve not read the news release.

    https://www.globenewswire.com/news-release/2021/09/30/2306460/13009/en/Catalyst-Pharmaceuticals-Receives-Positive-Decision-from-Appeals-Court-That-Supports-Orphan-Drug-Exclusivity-for-Firdapse-for-LEMS.html

    It’s a very long, and fractious story, getting from what was 3,4 Diaminopyridine to the brand name drugs, Firdapse and Ruzurgi.

    Here’s a bit from my perspective. I was diagnosed in January, 2018. Early on, getting accepted into a few LEMS Facebook groups, everyone was talking about 3, 4 Dap. It wasn’t an FDA approved drug, so for me, it was all just talk. But others were getting it in the U.S. from Jacobus Pharmaceuticals, I think classified as a study drug. The best part, those who got in on that, got it FREE. Yes, free, is quite the word in the drug world.

    Soon I called Jacobus Pharmaceuticals myself and at one time or another talked to Laura Jacobus and another person, several times. They were very nice, and set me up with the large amount of paperwork to get into their deal and get 3,4 Dap for myself. Unfortunately, I could never get my neurologist to do the paperwork and get me on it. I made several tries with her, but could never get her off “high center” to do it. I’ve heard several stories of people wanting on the drug, but unable to get paperwork accomplished. Let me remind you dear reader, if you’re still with me, there was NO FDA approved drug for LEMS.

    By a very fortuitous turn of events, I got myself into the early release program from Catalyst Pharmaceuticals in late September, 2018. I saw their study doctor, and in return received what would be named Firdapse, free (there’s that word again) during the trial. Now I was on the drug everyone was talking about, a derivative of 3, 4 Dap.

    On November 28, 2018 Firdapse was approved in the U.S. by the FDA. Catalyst began transitioning those of us on the study drug to the actual channels for commercial distribution. The drug was no longer going to be free. I understood this and expected it. Rare disease drugs are notoriously expensive due to the cost of trials and bringing the drug to market, one which by its very nature, is rare. LEMS is VERY rare.

    From my perspective, the good news was, now, any physician in the U.S. could prescribe 3, 4 Dap (as either Firdapse, or later Ruzurgi), and it was available. I accomplished my objective from early 2018 to reality in September and now it was available for any LEMS patient going forward by prescription. To me, this was a victory for patients.

    There’s much more to this story, but I’ll stop mine here for now.
    How did you get 3, 4 Diaminopyridine? I’d like to hear.

    price-wooldridge replied 2 years, 6 months ago 2 Members · 2 Replies
  • 2 Replies
  • joanne-evans

    Member
    October 9, 2021 at 5:11 pm

    I first received ruzurgi from a distributor in the USA. It was sent to me through my neurologist request. It was at a cost manageable as it wasn’t covered in Canada. Then I heard from the pharmacy in Ontario that the Supreme Court of Canada stopped the distribution. Firdapse became another option. I have been on it now for about 6 months and haven’t had to pay under a special warrant. Unfortunately this has come to an end. The cost will be prohibitive for me to continue.
    I’m extremely depressed and wonder what’s my next step. I am trying to get an appointment with a neurologist. Perhaps there is something else I can take. It is not an approved drug under our insurance.

  • price-wooldridge

    Member
    October 9, 2021 at 6:58 pm

    @joanneevans Hi Joanne! First, and it seems you have this already in process, contact your neurologist regarding amifampridine (Firdapse), its ongoing availability and alternatives. The distributor for Firdapse is KYE Pharmaceuticals in Canada. I don’t have their contact info. You may wish to reach out to them regarding Firdapse availability in Canada for you. In the U.S. Catalyst Pharmaceuticals has a robust patient assistance program for payments and insurance. Because you were receiving it in the U.S. I’m going to give you their Patient Assistance Program phone number. You may wish to reach out to them, and determine if they can help or how best for you to proceed.
    To talk with a Care Coordinator, call 1-833-422-8259. Service hours are Monday through Friday, 7:00 AM – 6:00 PM (Central Time), U.S.
    Please keep us posted as the landscape on amifampridine distribution in the U.S. and Canada is in flux.

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