Safety of Patisaran LNP
For those looking in on LNP safety here for gene therapy is some safety validation from our old LNPs used in ALNY Patisaran:
In the Global OLE Study, patients were treated with patisiran (overall mean
12 months) for an additional 11 or 21 months (APOLLO patisiran and Phase 2
patisiran, respectively) or newly treated for 10 months (APOLLO placebo), representing 211 patient-years and 3,506 doses.
Twenty-five patients, originally in the Phase 2 study, have now received patisiran for up to four years.
As of December 1, 2017, 25 patients (11.8 percent) have received over 269 infusions at home. One patient had a mild IRR at home that did not require treatment.
The safety profile of all patients in the Global OLE (N=211) was consistent with that observed in APOLLO, with infusion-related reactions (IRRs, 10.4 percent) representing the most common drug-related AE. There were two patients with reports of drug-related SAEs; one with phlebitis secondary to drug extravasation at the infusion site, cellulitis and hypotension, and one with abdominal discomfort, with the latter resulting in withdrawal from study.