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

  • I was on Ruzurgi for about 4 years before I was forced to take firdapse. I have been desperately trying to get back to Ruzurgi because I felt like I was weaker on firdapse which was confirmed when I went to my annual doctor visit with my neuro and he said I had lost 30% of my strength since being on Firdapse. I actually knew that before my doctor told me, but atleast I confirmed my belief. It is really difficult to lead a “normal” life when I am on firdapse. When I was on Ruzurgi, I was just about back to normal. I am fighting to obtain Ruzurgi so I can once again lead my best life possible.

  • Ed is far from dumb. He has researched and written multiple articles on this subject.

    He speaks to all the stakeholders and LEMS patients themselves.

    Have some compassion for people living with LEMS and the consequences many have suffered rather than lashing out in ignorance. It’s not just people’s portfolios at stake here.

  • As a Global LEMS advocate for 14 years who has been connected with hundreds of LEMS patients in both support forums and personally, I can attest to the multitude of harrowing accounts of the negative Firdapse experience as shared with me, not just in the US but also in parts of Europe.

    I have seen this situation unfold from beginning to the present day and it has been utterly devastating to watch.

    There was never any need to fix something which was not broken. There was nothing unstable about Jacobus DAP and hundreds of LEMS patients were the benefactors of this drug and the altruism of the company that made it and GAVE it to patients for 27 years – with the consent of the FDA on compassionate grounds.

    There was no issue in accessing DAP – there a few forms to fill out but so what?

    We were told it would be so much easier to get Firdapse but it was actually harder because patients then had to fight with their insurance companies and apply to assistance funds to get approved! How is ALL OF THAT – in anyone’s estimation – better or easier? And then for so many to suffer these adverse affects?

    And please tell me how there are grounds for a lawsuit against the FDA when Catalyst publicly states that have not been affected by the approval of Ruzurgi?

    Adding phosphate salt to an existing, effective drug and then and charging $375,000 per patient per year is neither altruistic nor moral.

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