Pasadena, USA | May 1, 2026
Arrowhead Pharmaceuticals has received approval from Australia’s Therapeutic Goods Administration (TGA) for REDEMPLO® (plozasiran), a first-in-class siRNA-based therapy, expanding global access for patients with familial chylomicronemia syndrome (FCS). This regulatory milestone highlights the growing impact of RNA interference (RNAi) therapeutics in treating rare metabolic diseases and reinforces the global expansion strategy for innovative gene-silencing medicines.
TGA Approval Marks Expansion of RNAi Therapeutics
The TGA approval authorizes REDEMPLO for use as an adjunct to diet in adult patients with genetically confirmed or clinically diagnosed FCS, a rare and severe disorder characterized by extremely elevated triglyceride levels and high risk of acute pancreatitis. This makes REDEMPLO the first and only approved treatment for FCS in Australia, addressing a significant unmet medical need where traditional lipid-lowering therapies often fail. The approval follows prior authorizations in the United States, Canada, and China, with a positive recommendation from European regulators, demonstrating the therapy’s strong global regulatory momentum.
Phase 3 Data Demonstrates Significant Clinical Benefit
The approval is supported by results from the Phase 3 PALISADE trial, a randomized, placebo-controlled study conducted across 39 global clinical sites, including multiple centers in Australia. The study met its primary and key secondary endpoints, showing a median triglyceride reduction of 80% from baseline, compared to a 17% reduction in the placebo group.
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Additionally, REDEMPLO significantly reduced the incidence of acute pancreatitis by 83%, a critical outcome given the life-threatening nature of the disease. These results highlight the therapy’s robust efficacy in managing severe hypertriglyceridemia and preventing disease complications, reinforcing its clinical value in rare disease management.
Targeted RNAi Mechanism Enables Precision Therapy
REDEMPLO leverages Arrowhead’s proprietary TRiM™ platform, utilizing small interfering RNA (siRNA) technology to silence the gene responsible for producing apolipoprotein C-III (apoC-III), a key regulator of triglyceride metabolism. By reducing apoC-III levels, the therapy enhances triglyceride clearance and significantly lowers circulating lipid levels, offering a targeted and sustained therapeutic approach.
The drug is administered via subcutaneous injection once every three months, improving patient adherence and convenience compared to traditional therapies. This mechanism represents a major advancement in precision medicine, where gene silencing enables direct intervention at the molecular level.
Safety Profile and Rare Disease Impact
REDEMPLO demonstrated a manageable safety profile, with the most common adverse events including hyperglycemia, headache, nausea, and injection site reactions. The benefit-risk profile supports its use in a patient population with limited treatment options and high disease burden, where untreated FCS can lead to recurrent pancreatitis, chronic complications, and reduced quality of life. The approval also reflects increasing regulatory support for orphan drugs and breakthrough therapies, particularly those addressing rare and underdiagnosed conditions.
Advancing Global Access and Future Development
With this approval, Arrowhead continues to expand access to RNAi-based therapies across global markets, while advancing ongoing Phase 3 clinical programs for related lipid disorders. The success of REDEMPLO underscores the potential of RNA interference technology to transform treatment paradigms for metabolic and genetic diseases, paving the way for future innovations in gene-silencing therapeutics.
Source: Arrowhead Pharmaceuticals press release
