Alnylam Presents New Long-Term Results from Global Open-Label Extension Study of ONPATTRO (patisiran) at the European Academy of Neurology Virtual Congress 2020 - Seite 2

For APOLLO placebo patients subsequently treated with patisiran for 24 months in the OLE study, neuropathy progression was also notably halted and QOL improved. However, these patients had experienced rapid progression while on placebo in the APOLLO study and did not return to their baseline scores, highlighting the importance of patients starting treatment with patisiran early.

The long-term safety profile of patisiran was consistent with that observed and previously reported in the APOLLO Phase 3 study and the Phase 2 OLE study.

ONPATTRO in Patients with Disease Progression Post-Orthotopic Liver Transplant

In addition, data were presented from an interim analysis of the Phase 3b open-label study conducted across several European countries to evaluate the safety, efficacy and pharmacokinetics (PK) of patisiran in patients with hATTR amyloidosis with disease progression after receiving an orthotopic liver transplant (OLT). Historically, OLT has been used to slow disease progression in patients with early stages of hATTR amyloidosis; however, some patients experience disease progression after the transplant due to continued amyloid deposition of wild-type TTR on top of existing amyloid deposits in tissues.

Twenty-three patients who showed disease progression post-OLT (based on an increase in polyneuropathy disability [PND] score) received patisiran infusion (0.3 mg/kg) every three weeks. After 3 weeks of patisiran treatment, the mean reduction from baseline in serum TTR levels was 81.9 percent. At the time of interim safety analysis (data cutoff as of December 9, 2019), the safety profile of patisiran in this Phase 3b study was consistent with that observed and previously reported in the APOLLO Phase 3 study. The safety, efficacy, and PK of patisiran treatment post-OLT will be further investigated in this ongoing study.

To view the data presented at EAN, please visit www.alnylam.com/capella.

ONPATTRO (patisiran) lipid complex injection Important Safety Information

Infusion-Related Reactions

Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO. In a controlled clinical study, 19 percent of ONPATTRO-treated patients experienced IRRs, compared to 9 percent of placebo-treated patients. The most common symptoms of IRRs with ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea, and headache.