ASH: Sanofi un­corks late-stage da­ta for RNAi drug fi­tusir­an in he­mo­phil­ia, with all eyes on safe­ty pro­file

Sanofi’s fi­tusir­an has had a rough road in he­mo­phil­ia, weath­er­ing clin­i­cal holds and pro­gram halts tied to its lin­ger­ing safe­ty woes. Now, the drug is near­ing the fin­ish line with late-stage da­ta in hand, but will those same safe­ty con­cerns slam the brakes on the pro­gram de­spite its deliri­ous­ly ef­fec­tive re­sults?

Fi­tusir­an, an RNAi drug de­signed to si­lence the gene that over­pro­duces a pro­tein re­spon­si­ble for clot­ting sup­pres­sion, sig­nif­i­cant­ly re­duced the an­nu­al­ized rate of bleed­ing over on-de­mand fac­tor ther­a­py in he­mo­phil­ia A/B pa­tients with­out pre­ex­ist­ing fac­tor in­hibitors in the blood, ac­cord­ing to late-break­ing da­ta pre­sent­ed Tues­day at #ASH21.

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