Io­n­is touts PhI­II suc­cess for rare dis­ease drug amid com­pa­ny's push to com­mer­cial­ize drugs in­de­pen­dent­ly

Io­n­is Phar­ma­ceu­ti­cals’ hered­i­tary an­gioede­ma treat­ment helped cut the rate of at­tacks as­so­ci­at­ed with the rare ge­net­ic dis­ease in a Phase III study.

Peo­ple with hered­i­tary an­gioede­ma, or HAE for short, ex­pe­ri­ence episodes of se­vere swelling in var­i­ous parts of the body. In a piv­otal study with 91 par­tic­i­pants, those who re­ceived donidalors­en every four or eight weeks had a low­er rate of at­tacks com­pared to those who re­ceived place­bo, Io­n­is an­nounced Mon­day.

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