When a drug for a rare disease gets FDA approval

Yesterday was such an exciting day for those of us who suffer from Lambert-Eaton Myesthenic Syndrome. Catalyst Pharmaceuticals gained approval for the first approved treatment for LEMS, Firdapse.

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What does this mean when you have a rare disease?

This means doctors will have heard of your rare illness that affects only 1 in 1 million people. Up until now, many doctors had not even heard of it. I’ve been the educator for most of my providers as they have said themselves when asked: “Well, I’ve only read a paragraph about it in med school.”

This means that neurologists will consider LEMS as the possible cause to the unidentified neuromuscular symptoms a LEMS patient is experiencing much faster. Many patients, including myself, went through years of debilitating symptoms prior to diagnosis and treatment. LEMS patients should no longer have to often times wait years for diagnosis and will be able to have a treatment prescribed. Trials are hard to access and often times unknown. Thank goodness the trials were successful so that all LEMS patients can benefit!

This means that our invisible illness is now visible in the form of an FDA approved drug to treat our illness and give us our quality of life back. Prior to treatment, some LEMS patients lose the ability to walk, to swallow, to breath. Some it’s not as severe, but it consists of years of debilitating, unexplained weakness and exhaustion. Some, like me, go undiagnosed for so long that the disease progresses to severe prior to diagnosis thus is harder to manage and much more intensive treatment is needed.

This approval opens up the world of diagnosis and treatment of LEMS to everyone, not just those with doctors who won’t stop until they find the answer to what is causing your symptoms. Some doctors stop. Some doctors think it’s all in your head. Sometimes you wonder yourself if it IS in your head. Once diagnosed, the relief a patient feels for knowing what is behind the weakness and numbness is amazing. It doesn’t matter that you have this very rare illness that most doctors will never see. At least there’s a reason behind the symptoms! Now, we finally have an FDA approved treatment, Firdapse!

There are always nervous questions about insurance coverage and gaining access to the medication once approved. However, I have faith that the work has been done for patients to access Firdapse through their just announced patient support program, Catalyst Pathways™ , because if we as patients can’t access Firdapse, then why would Catalyst Pharmaceuticals have put so much work into gaining approval? Our access will equal their success.

This is a brand new chapter in the very small book of LEMS. We are few but we are mighty. And now that a drug to treat our very rare disease is approved, ALL LEMS patients will have a weapon in their arsenal against this debilitating neuromuscular disease.

Dawn DeBois

About Dawn DeBois

Florida born and Maine grown, my life has been atypical. My childhood was full of loss and severe physical pain. Both emotional and physical stress during childhood has been found to contribute to autoimmune disease. My first autoimmune diagnosis was at the age of 28, which has led to juggling multiple autoimmune diseases (Hashimoto’s, Fibromyalgia, Ankylosing Spondylitis, Psoriatic Arthritis and most recently LEMS- Lambert Eaton Myasthenic Syndrome). I am officially now classified as having “Multiple Autoimmune Syndrome.” You know what they say, go big or go home!