Last February, Barbara Moore switched to a new medicine to combat a rare neuromuscular disease that had plagued her for nearly a quarter of a century.

She had not planned to do so, but Moore had no choice: An effective treatment she had been taking all those years was forced off the market. In late 2018, Catalyst Pharmaceuticals (CPRX) was granted exclusive marketing rights to sell its new drug, called Firdapse, after the Food and Drug Administration approved the medicine. The move precludes any competition for seven years.

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  • I see that more and more people have negative side effects form Firdapse. Not only in the US, but global users too. I live in Europe in the Netherlands and I use 3,4 DAP since I was 28. In 2014 The health insurance said there where no ingredients anymore to make 3,4 DAP, so I was forced to use FIRDAPSE.

    What a set back I had and too many side effects, such as; Shortness of breath or trouble breathing, a heart that suddenly beated 38 times per minute, I got a pacemaker for that., Muscle spasms, stomach pain, Nausea,Diarrhea, Headache , Increased liver enzymes, Back pain, High blood pressure. I couldn’t walk anymore and barely speak or swallow. Plus vomitting and being constant dizzy.

    After a while my doctor contacted the health insurance that i had no other choiche to go back to my 3,4 DAP. It was a miracle. After a few days I could do the normal things again, like better walking and talking and swallow. So i hope I can use 3,4 DAP forever. Or when there will be a better solution. But no more Firdapse for me.

  • Not every drug works the same way with every patient. Fidapse was tested successfully in clinical trials so i am not exactly sure what the motivations of some here may be!

    • Kerem,
      They possibly neglected to mention that their “trials “ were skewed by the fact that if patients experienced huge side effects, they were immediately removed from the study and the offending drug demanded back.

    • We are talking about a tiny patient group within which numerous individuals are reporting devastating effects. I personally am aware of (and in contact with) 39 such people. With only a few hundred people on Firdapse, does that seem problematic to you? We are not talking about one in a million here.

      What is your motivation? What’s at stake for you here?

  • I find it laughable that Catalyst believes that offering their drug to patients for a copay of $10 somehow makes everything ok.

    Their drug is inferior in every way to it’s predecessor Ruzurgi and offering it for $10 does not erase that fact. A good price on arsenic does NOT make it safer to take.

    They can make public statements all they like about how well Lems patients love their drug, and they have to say that to keep investors happy, but here in the real world we know that is all PR hype.

    Ruzurgi is the one that actually works.

  • Unfortunately many LEMS patients are battling multiple health issues. They and their doctors often spend years, sometimes through painful trial and error, working out a very individualized drug cocktail. Any alteration of the recipe, no matter how small, can have devastating effects. Firdapse and Ruzurgi are not the same drug. The people who were taking Ruzurgi when it was called DAP, saw it as their miracle drug which greatly improved their quality of life. As many of them see it, Catalyst took away a drug that was working for them and replaced it with an inferior substitute. Catalyst’s arrival and the way it handled it’s launch of Firdapse created a wedge within the tiny LEMS community. We felt like we had to choose a side and began fighting with each other. It did damage to relationships within the support groups that may never be repaired. LEMS feeds on stress. With FDA approval of Ruzurgi, LEMS patients can now choose the drug that works best for them and get back to living the best life possible.

  • This is not the first instance of FDA ‘jumping’ the gun in favor of a new upstart possibly with more dollars in lobbying. How sad for those patients. The dollar wins with the FDA not patient health. Thankfully I use essential oils successfully for arthritis and other aging issues There are no contraindications and in Pharmacopoeia for thousands of years

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