Al­ny­lam shores up en­thu­si­asm for givosir­an with ear­ly dataset ahead of cru­cial PhI­II read­out

In the run-up to top-line re­sults from a cru­cial Phase III for its sec­ond RNAi drug, Al­ny­lam has so­lid­i­fied an ear­ly set of safe­ty and ef­fi­ca­cy num­bers for givosir­an’s da­ta pack­age — part of a rolling sub­mis­sion at the FDA.

Re­sults from the Phase I, which we first heard about last April, sug­gest that month­ly in­jec­tions of givosir­an re­duced the num­ber of at­tacks in pa­tients with acute he­pat­ic por­phyr­ia by up to 79%, ac­cord­ing to a new pa­per in the New Eng­land Jour­nal of Med­i­cine. The us­age of hemin, an in­fu­sion cur­rent­ly used to treat these at­tacks, al­so dropped by 83% com­pared to place­bo, the com­pa­ny added.

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