Al­ny­lam sets up 3rd po­ten­tial RNAi ap­proval with a show­case of pos­i­tive lumasir­an da­ta

A week af­ter nab­bing a pri­or­i­ty re­view, Al­ny­lam has re­leased piv­otal da­ta for their lat­est late-stage ex­per­i­men­tal drug — set­ting the stage for the RNA-tar­get­ing biotech to po­ten­tial­ly win their 3rd FDA ap­proval in as many years.

In the 39-per­son tri­al, pa­tients with an ul­tra-rare liv­er con­di­tion PH1 (pri­ma­ry hy­per­ox­aluria type 1) were giv­en ei­ther place­bo or the Al­ny­lam drug lumasir­an. Pa­tients with PH1 over­pro­duce a nat­ur­al meta­bol­ic end-prod­uct, uri­nary ox­alate, po­ten­tial­ly lead­ing to kid­ney stones and in many cas­es kid­ney fail­ure at a young age, along with dam­age across the body.

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