Cat­a­lyst Phar­ma's LEMS drug Fir­dapse wins FDA nod, as reg­u­la­tors keep the ap­proval en­gine run­ning at record speed

Turns out, a web er­ror by a com­pa­ny con­trac­tor that tipped off in­vestors Cat­a­lyst Phar­ma­ceu­ti­cals’ $CPRX Fir­dapse had won ap­proval ear­li­er in the week of­fered alert traders a quick score. The for­mal no­tice that the FDA green-light­ed the treat­ment for the rare neu­ro­mus­cu­lar dis­ease came through Wednes­day evening.

The drug is to be used in pa­tients with Lam­bert-Eaton Myas­thenic Syn­drome (LEMS), a rare au­toim­mune dis­ease that af­fects about 1 in every 300,000 peo­ple. In pa­tients with LEMS, the body’s im­mune sys­tem at­tacks the neu­ro­mus­cu­lar junc­tion (the con­nec­tion be­tween nerves and mus­cles) and dis­rupts the abil­i­ty of nerve cells to send sig­nals to mus­cle cells, caus­ing weak­ness and fa­tigue that typ­i­cal­ly cul­mi­nates in dif­fi­cul­ties with dai­ly func­tion­ing. LEMS is of­ten as­so­ci­at­ed with oth­er au­toim­mune dis­eases, and com­mon­ly oc­curs in pa­tients with small cell lung can­cer.

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