Arrowhead Pharmaceuticals secures Therapeutic Goods Administration approval for REDEMPLO (plozasiran) in familial chylomicronemia syndrome, backed by Phase 3 data showing 80% triglyceride reduction and reduced pancreatitis risk.
Written by: Farha Farheen, Pharm D
Reviewed By: Pharmacally Editorial Team
Arrowhead Pharmaceuticals has received approval from the Therapeutic Goods Administration (TGA) for REDEMPLO® (plozasiran) as an adjunct to diet to reduce triglyceride levels in adults with familial chylomicronemia syndrome (FCS) who have not responded adequately to standard therapies. REDEMPLO is the first approved therapy for FCS in Australia and is indicated for both genetically confirmed and clinically diagnosed patients.
The approval follows prior authorizations in the United States, Canada, and China, along with a positive opinion from the European Medicines Agency supporting marketing authorization.
The approval is supported by results from the Phase 3 PALISADE trial (NCT05089084), a randomized, double-blind, placebo-controlled study conducted across 39 global sites. Treatment with 25 mg plozasiran resulted in a median triglyceride reduction of 80% from baseline compared with 17% with placebo, and the pooled dose groups showed 83% lower odds of acute pancreatitis compared with placebo (4% vs 20%).
Significant reductions in apoC-III and other key secondary endpoints were also observed. The therapy is administered as a subcutaneous injection once every three months.
Familial chylomicronemia syndrome is a rare and underdiagnosed condition, affecting approximately 1 to 13 individuals per million globally. Plozasiran is a small interfering RNA (siRNA) therapy developed using Arrowhead’s TRiM™ platform that targets and silences the messenger RNA encoding apolipoprotein C-III (apoC-III), a key regulator of triglyceride metabolism. By reducing apoC-III levels, the therapy enhances triglyceride clearance and leads to sustained reductions in circulating triglycerides.
The most common adverse event reported was hyperglycaemia (12.8%), with other events including headache (6.8%), nausea (4.7%), and injection site reactions (4.7%).
Christopher Anzalone stated that the approval reflects the strength of the clinical data and the potential of the company’s RNA interference platform to address difficult-to-treat diseases.
Gerald F Watts noted that the results demonstrate substantial and sustained triglyceride reductions and represent a meaningful advancement for patients with limited treatment options.
Familial chylomicronemia syndrome is a genetic disorder characterized by extremely high triglyceride levels, often exceeding 10 mmol/L (880 mg/dL), which significantly increases the risk of acute and recurrent pancreatitis and contributes to substantial disease burden.
REDEMPLO has received multiple regulatory designations from the U.S. Food and Drug Administration, including Breakthrough Therapy, Fast Track, and Orphan Drug status, and has also been granted orphan designation by the EMA.
Plozasiran is also under evaluation in multiple Phase 3 studies for severe and broader hypertriglyceridemia populations.
Results from the PALISADE study have been presented at major scientific meetings and published in leading journals, including The New England Journal of Medicine and Circulation.
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About the Writer
Farha Farheen, Pharm.D, is a pharmacy professional with a strong interest in pharmacovigilance and clinical research. She has completed her Doctor of Pharmacy (Pharm.D) along with her internship as a Clinical Pharmacist. She has hands-on experience in adverse drug reaction (ADR) reporting, safety data documentation, and pharmacovigilance workflows, and is proficient in using VigiFlow. She is also a patent holder for an antibacterial formulation enriched with bioactive substances, granted by the German Patent and Trademark Office.