Genfit, Ipsen tout pivotal trial readout for rare liver disease, head toward FDA and EMA submissions

Gen­fit and Ipsen an­nounced Fri­day morn­ing that their ex­per­i­men­tal treat­ment elafi­bra­nor helped pa­tients with pri­ma­ry bil­iary cholan­gi­tis, a rare liv­er dis­ease, see a de­crease in im­por­tant bio­mark­ers of dis­ease pro­gres­sion.

In a Phase III tri­al with 161 pa­tients, just over half of those who re­ceived 80 mg of elafi­bra­nor met the pri­ma­ry end­point com­pared to on­ly 4% of pa­tients on place­bo. The end­point mea­sured cholesta­sis re­sponse — char­ac­ter­ized by de­creas­es in a liv­er en­zyme called ALP and de­creas­es in biliru­bin. In­creased lev­els of ei­ther usu­al­ly point to liv­er in­jury.

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