Abstract from Alnylam:
Initial results from Part C, which is a randomized, double-blind, placebo-controlled (3:1, drug:placebo) study in patients with acute intermittent porphyria (AIP) experiencing recurrent attacks, showed robust and durable lowering of aminolevulinic acid (ALA) and porphobilinogen (PBG), the toxic heme intermediates that are believed to mediate porphyria symptoms and acute attacks.
As of the data transfer on November 7, 2016, there were no drug-related serious adverse events (SAEs) reported in Cohorts 1-4. In Cohort 3, which remains blinded, one death was reported after the data transfer date due to acute pancreatitis, complicated by a pulmonary embolism; the death was considered to be unlikely related to givosiran or placebo by the investigator and the study’s Safety Review Committee.
Of note, increases in pancreatic enzymes and acute pancreatitis have been reported in the literature in patients with acute hepatic porphyria (Shen et al., Acta Neurol Taiwan, 2008;17:177-183; Shiraki et al., Nihon Rinsho, 1995;53:1479-1483).
In Cohorts 1 and 2, there were no discontinuations due to adverse events (AEs). Possibly or definitely related AEs reported in two or more cases were injection site reactions and myalgia; all of these events were mild. There were no other clinically significant changes in vital signs, electrocardiograms, clinical laboratory parameters, or physical examination.