The Food and Drug Administration just added an unexpected twist to a simmering controversy over a rare disease drug that earlier this year briefly became a poster child for high-priced medicines.

In a surprise move, the agency approved a medicine from Jacobus Pharmaceuticals, a small, family-run company, for treating a neuromuscular disorder called Lambert-Eaton myasthenic syndrome, or LEMS, for children ages 6 to 17. However, the approval potentially adds unforeseen competition for Catalyst Pharmaceuticals (CPRX), which only last November won an FDA endorsement to market its own treatment for adults.

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  • fun fact:

    Firdapse is 3,4-diaminopyridine, a patent free chemical compound that can be bought from chemicals suppliers at 100$/5g, good for about 150 daily doses.

  • Interesting workaround the exclusivity issue and pricing. But rarely do 2 companies have the exact same drug going up for NDA approval where exclusivity is an issue blocking the other from the market. (Recognizing it commonly happens with generics.) It will be interesting to see how this plays out.

  • Catalyst is the villain here. I have taken DAP for many years. It has been free from Jacobus, just have to pay for the shipping, which the hospital I get if from is charging. I have every faith that Laura Jacobus will price it fairly. It might even be less than the current shipping cost because it has to stay refrigerated and the packaging is extensive. Many people with LEMS and diseases with similar symptoms, who are also taking DAP, are rejoicing right now.

    • fun fact:
      Firdapse is 3,4-diaminopyridine, a patent free chemical compound that can be bought from chemicals suppliers at 100$/5g, good for about 150 daily doses.

  • May I observe that it is ,,, bemusing (?) for the Jacobus CEO to say they haven’t thought much about pricing, if there goal for years has been to sell it? That said, “free” is definitely out the window when you consider $60 million of carried cost plus say $15 million to come?
    Let’s consider 1,000 patients (why not use this number?) against $75 million …. minimum cost $75K without including any profit or reserve. Let’s say $100K – a big jump from compassionate use. We shall have to see how this play out.

    • To Observer09: Your assumption is that she makes it all back in one year – using your per person per year numbers, a 10-year payback would bring it to $10K per year, no gimme but well within the cost of many specialty drugs (now if the government would help some …. ).

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