Cat­a­lyst shares jazzed by a new round of pos­i­tive re­sults for a rare neu­ro­mus­cu­lar dis­ease

Cat­a­lyst Phar­ma­ceu­ti­cals says it scored promis­ing da­ta from a tiny mid-stage study of its lead drug Fir­dapse in an ul­tra-rare neu­ro­mus­cu­lar dis­ease, set­ting up a shot at a piv­otal study that can aim at an FDA ap­proval. But this time the biotech — which has some cred­i­bil­i­ty prob­lems af­ter an em­bar­rass­ing set­back last year — wants some feed­back from the agency be­fore set­ting up a time­line.

A year ago, the FDA sent Cat­a­lyst back to the draw­ing board with Fir­dapse, hand­ing the biotech a rare refuse-to-file let­ter for the drug as a treat­ment for Lam­bert-Eaton myas­thenic syn­drome (LEMS), a rare neu­ro­mus­cu­lar con­di­tion, lat­er say­ing it would need to run a new study and gath­er more da­ta ahead of an ap­proval. The FDA has hand­ed the com­pa­ny a break­through ther­a­py des­ig­na­tion for that in­di­ca­tion.

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