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.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER