Catch­ing Cat­a­lyst Phar­ma by sur­prise, FDA ap­proves Ja­cobus LEMS drug for pe­di­atric pa­tients — based on adult da­ta

When Cat­a­lyst Phar­ma­ceu­ti­cals’ drug for Lam­bert-Eaton myas­thenic syn­drome (LEMS) — a rare, au­toim­mune dis­or­der that af­fects the con­nec­tion be­tween nerves and mus­cles — was ap­proved last year, car­ry­ing a $375,000 an­nu­al price tag, some pa­tients were not ex­act­ly thrilled. Hun­dreds of pa­tients had been able to ac­cess a sim­i­lar drug from com­pound­ing phar­ma­cies for a frac­tion of the cost, or from a small, fam­i­ly-run New Jer­sey-based com­pa­ny called Ja­cobus Phar­ma­ceu­ti­cals for free, as part of an FDA-rat­i­fied com­pas­sion­ate use pro­gram.

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