Io­n­is plans NDA fil­ing for first in­de­pen­dent drug af­ter pos­i­tive da­ta read­out in rare ge­net­ic dis­or­der

Io­n­is is an­oth­er step clos­er to the launch of its first in­de­pen­dent drug, tout­ing pos­i­tive Phase III da­ta for olezarsen in pa­tients with a rare ge­net­ic dis­or­der called fa­mil­ial chy­lomi­crone­mia syn­drome (FCS).

Io­n­is said Tues­day it has plans to file for an NDA in ear­ly 2024 and for ap­provals in the EU af­ter olezarsen hit its pri­ma­ry end­point in the BAL­ANCE study: a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion in triglyc­eride lev­els af­ter six months on 80 mg of the drug com­pared to place­bo, earn­ing a p-val­ue of 0.0009. A key sec­ondary end­point al­so showed promise, with olezarsen re­duc­ing acute pan­cre­ati­tis events by 100%. In the place­bo arm, there were 11 in­stances of pan­cre­ati­tis.

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