Io­n­is says it gath­ered promis­ing PhI­II da­ta for a top rare dis­ease drug, but se­ri­ous safe­ty ques­tions re­main

Io­n­is Phar­ma­ceu­ti­cals $IONS says that its Phase III study of in­ot­ersen (IO­N­IS-TTR­Rx) hit both pri­ma­ry end­points in pa­tients with rare cas­es of fa­mil­ial amy­loid polyneu­ropa­thy (FAP). But in­ves­ti­ga­tors al­so spelled out just how dan­ger­ous this drug can be, cloud­ing its fu­ture with reg­u­la­tors.

The FDA once put this drug on hold, caus­ing Glax­o­SmithK­line to edge away from their col­lab­o­ra­tion on the drug. But GSK still has an op­tion to grab mar­ket­ing rights on the ther­a­py, as Io­n­is push­es ahead with plans to file for an ap­proval.

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