FDA mo­tions Al­ny­lam's lumasir­an in­to a PhI­II short track, set­ting up a shot at an OK in '20

Al­ny­lam $AL­NY may be go­ing so­lo on the de­vel­op­ment of rare dis­ease drug lumasir­an af­ter Sanofi took a pass on its op­tion, but the FDA has come through with a ma­jor as­sist. The com­pa­ny an­nounced to­day that reg­u­la­tors have agreed on a Phase III study de­sign that would ac­cel­er­ate — should all go well — its jour­ney to ap­proval.

For the tri­al de­signed to test lumasir­an in the ul­tra-rare pri­ma­ry hy­per­ox­aluria type 1 (PH1), the new­ly reached align­ment al­lows Al­ny­lam to adopt a pri­ma­ry end­point based on a bio­mark­er: the re­duc­tion of uri­nary ox­alate at six months. The FDA has al­so sanc­tioned a study size of around 25 pa­tients with PH1.

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