Arrowhead Pharmaceuticals has announced that Health Canada has granted a Notice of Compliance (NOC) for REDEMPLO (plozasiran), a novel small interfering RNA (siRNA) therapy designed to lower dangerously high triglycerides in adults with familial chylomicronemia syndrome (FCS) when standard treatments fail.

 

FCS is a rare, severe disorder that can cause triglyceride levels 10 to 100 times above normal, dramatically increasing the risk of acute, recurrent, and potentially fatal pancreatitis. The condition is underdiagnosed, affecting 1–13 people per million globally, though rates are roughly 100 times higher in parts of French Canada.

 

REDEMPLO is the first and only Health Canada-approved siRNA therapy for FCS, offering patients the convenience of a subcutaneous injection once every three months at home. The approval follows recent U.S. authorization as Arrowhead works to expand global access to this life-changing treatment.

 

“We are thrilled to start the new year with Health Canada’s approval of REDEMPLO as a new treatment option for Canadians living with genetic or clinical FCS,” said Christopher Anzalone, President and CEO of Arrowhead Pharmaceuticals. 

 

“Arrowhead continues to drive RNAi innovation with our TRiM platform, which is now capable of delivering siRNA to multiple cell types throughout the body and potentially treat a growing array of diseases. As we expand our commercial capabilities, we aim to bring these new treatments to the patients who need them.”

 

REDEMPLO works by targeting APOC3 mRNA to reduce triglycerides. Health Canada’s approval was supported by results from the Phase 3 PALISADE trial, which enrolled adults with clinically diagnosed or genetically confirmed FCS across 39 sites worldwide, including five in Canada. 

 

The study demonstrated significant, durable triglyceride reductions—25 mg of REDEMPLO lowered median triglycerides by 80% over 12 months, compared to 17% in placebo patients—while also lowering the incidence of acute pancreatitis. Most common side effects included hyperglycemia, nausea, and injection site reactions.

 

REDEMPLO’s approval marks a milestone for patients, caregivers, and healthcare providers seeking a convenient, home-based therapy for FCS.

 

“For more than two decades, we have hoped along with the FCS community the next therapy would truly help end the excruciating pain and unpredictability of this very challenging condition—and thanks to the unrelenting commitment of researchers and scientists, we believe the time has arrived,” said Durhane Wong-Rieger, President & CEO of Canadian Organization for Rare Disorders.