John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

With lumasir­an on the FDA's doorstep, Al­ny­lam reads out new PhI­II da­ta in PH1

Just over a month away from its De­cem­ber PDU­FA date, Al­ny­lam flaunt­ed new da­ta from two Phase III stud­ies to back lumasir­an in pri­ma­ry hy­per­ox­aluria type 1 (PH1), a rare liv­er con­di­tion.

The Cam­bridge, MA-based biotech snagged a pri­or­i­ty re­view for the can­di­date back in June, and got pos­i­tive feed­back from the EMA’s Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use just last week. Lumasir­an us­es RNA in­ter­fer­ence (RNAi) to si­lence the gene for gly­co­late ox­i­dase, an en­zyme used in the pro­duc­tion of ox­alate.

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