Australia Approves First FCS Treatment REDEMPLO by Arrowhead

Arrowhead Pharmaceuticals, Inc. has announced that Australia’s Therapeutic Goods Administration (TGA) has approved REDEMPLO® (plozasiran), marking the first authorized treatment in the country for patients with familial chylomicronemia syndrome (FCS), a rare and potentially life-threatening genetic disorder.

The approval allows REDEMPLO to be used alongside dietary management to reduce triglyceride levels in adult patients whose condition has not been adequately controlled with standard therapies. FCS is characterized by extremely elevated triglyceride levels, which significantly increase the risk of recurrent and potentially fatal pancreatitis. Despite its severity, the disease remains underdiagnosed and is estimated to affect between one and 13 individuals per million globally.

With this decision, Australia joins the United States, Canada, and China in approving REDEMPLO, while the European Medicines Agency has also issued a positive opinion recommending its authorization. The therapy is approved for both genetically confirmed and clinically diagnosed adult FCS patients, broadening access to treatment.

REDEMPLO is based on small interfering RNA (siRNA) technology and works by silencing the production of apolipoprotein C-III (apoC-III), a protein that plays a central role in regulating triglyceride metabolism. By reducing apoC-III levels, the drug enhances the body’s ability to break down and clear triglycerides from the bloodstream.

The TGA’s approval was supported by results from the Phase 3 PALISADE clinical trial, a global, randomized, placebo-controlled study involving patients with FCS. The trial demonstrated significant clinical benefits, including a median reduction in triglyceride levels of approximately 80% among patients receiving REDEMPLO, compared to a 17% reduction in the placebo group. Additionally, the treatment was associated with an 83% lower likelihood of acute pancreatitis events.

REDEMPLO is administered as a subcutaneous injection once every three months, offering a relatively convenient dosing schedule for patients managing a chronic condition. Safety data indicated that the most common side effect was hyperglycaemia, along with mild reactions such as headache, nausea, and injection site reactions.

Company leadership highlighted the importance of the approval for patients with limited treatment options. Christopher Anzalone, President and CEO of Arrowhead, said the decision underscores the strength of the company’s RNA interference platform and its ability to deliver targeted therapies across multiple diseases.

Clinical experts also welcomed the development. Gerald F Watts of the University of Western Australia noted that patients with FCS face a lifelong burden of disease and that plozasiran represents a meaningful advancement in care, with the potential to significantly reduce triglyceride levels and associated complications.

The therapy has also received multiple regulatory designations from the U.S. Food and Drug Administration, including Breakthrough Therapy, Fast Track, and Orphan Drug status, reflecting its potential to address a high unmet medical need.

The approval of REDEMPLO marks a major step forward in the treatment of FCS and highlights the growing role of RNA-based therapies in tackling rare and complex diseases.