Arrowhead Pharmaceuticals has received regulatory clearance from Health Canada for REDEMPLO (plozasiran), marking a significant advance for patients living with familial chylomicronemia syndrome (FCS), a rare and severe genetic disorder characterized by extremely elevated triglyceride levels. Health Canada issued a Notice of Compliance authorizing REDEMPLO as an adjunct to diet to reduce triglycerides in adults with FCS who have not achieved adequate control with standard triglyceride-lowering therapies.
FCS is a debilitating and often underdiagnosed condition in which triglyceride levels can be 10 to 100 times higher than normal, placing patients at high risk of acute, recurrent, and potentially life-threatening pancreatitis. Globally, FCS is estimated to affect between one and 13 people per million, though prevalence is significantly higher in certain regions, including parts of French Canada. Patients often face strict dietary restrictions and limited therapeutic options, as conventional lipid-lowering drugs are typically ineffective.
REDEMPLO is a small interfering RNA (siRNA) medicine designed to address the underlying biology of the disease. It is the first and only siRNA therapy approved by Health Canada for FCS and can be self-administered at home as a subcutaneous injection once every three months. The approval follows the recent authorization of REDEMPLO in the United States and reflects Arrowhead’s broader strategy to expand global access to RNA interference–based therapies.
The Canadian approval is supported by results from the Phase 3 PALISADE study, a randomized, double-blind, placebo-controlled trial involving adults with either clinically diagnosed or genetically confirmed FCS. The study was conducted across 39 sites worldwide, including five in Canada. PALISADE met its primary endpoint and all key secondary endpoints, demonstrating significant reductions in triglyceride levels and APOC3, a key protein involved in triglyceride metabolism. Patients receiving 25 mg of REDEMPLO achieved a median triglyceride reduction of 80% over 12 months, compared with a 17% reduction in the pooled placebo group. The study also showed a lower numerical incidence of acute pancreatitis in the treatment arm. The most common adverse reactions included hyperglycemia, nausea, and injection site reactions.
REDEMPLO is built on Arrowhead’s proprietary Targeted RNAi Molecule (TRiM) platform and works by silencing the messenger RNA for APOC3, a validated therapeutic target in FCS. Notably, it is the first Health Canada–approved treatment studied in both genetically confirmed and clinically diagnosed patients, offering a broader evidence base for real-world use.
Patient advocacy groups and clinical investigators welcomed the decision. Representatives from the Canadian Organization for Rare Disorders highlighted the potential for the therapy to reduce disease unpredictability and improve quality of life for patients and families. Investigators involved in the PALISADE trial described the approval as a potential turning point for a community that has long had limited and ineffective treatment options.
Arrowhead noted that the pivotal study results were presented at major international cardiology meetings in 2024 and published in leading medical journals. The company added that it is developing patient support programs to help ensure access and appropriate use across Canada. REDEMPLO has also received multiple regulatory designations in the United States and Europe, underscoring its importance as a novel therapy for this rare disease.