-Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) announced that the Australian Therapeutic Goods Administration (TGA) has approved REDEMPLO® (plozasiran), a small interfering RNA (siRNA) medicine, as an adjunct to diet to reduce triglyceride levels for adult patients with familial chylomicronemia syndrome (FCS) for whom standard triglyceride lowering therapies have been inadequate. FCS is a severe, rare disease characterized by triglyceride levels that can be orders of magnitude higher than normal, leading to a substantially higher risk of developing acute, recurrent, and potentially fatal pancreatitis. FCS remains widely underdiagnosed and affects an estimated 1 to 13 people per million globally.

We are pleased that REDEMPLO is now the first-ever approved treatment for Australians living with genetic or clinical FCS

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REDEMPLO is the first and only medicine approved for use in the treatment of FCS in Australia, and is authorized for both genetically confirmed and clinically diagnosed adult patients with FCS. This approval by TGA follows approvals in the United States, Canada, and China, and a positive opinion recommending marketing authorization from EMA as Arrowhead continues its efforts to increase global access to care for people living with FCS.

“We are pleased that REDEMPLO is now the first-ever approved treatment for Australians living with genetic or clinical FCS,” said Christopher Anzalone, Ph.D., President and CEO of Arrowhead Pharmaceuticals. “This approval underscores the strength of the clinical data and the ability of our TRiM™ platform to develop targeted siRNA therapies to potentially reach multiple tissues and disease areas. We believe REDEMPLO could represent an important medicine for the FCS community in Australia, and we are working to bring this therapy to more patients as quickly as possible.”

Harnessing Arrowhead’s proprietary Targeted RNAi Molecule (TRiM™) platform, REDEMPLO is designed to silence the mRNA encoding apolipoprotein C-III (apoC-III). ApoC-III is a key regulator of triglyceride metabolism that inhibits triglyceride catabolism and clearance, resulting in elevated triglyceride levels. Individuals with genetic loss-of-function variants in APOC3 typically have markedly lower triglyceride levels and a reduced risk of atherosclerotic cardiovascular disease.

The Therapeutic Goods Administration approval was supported by clinical data from the Phase 3 PALISADE study, a randomized, double-blind, placebo-controlled trial in adults with clinically diagnosed or genetically confirmed FCS. The clinical study was conducted across 39 global sites, including 5 study locations in Australia. The PALISADE study met its primary endpoint and all multiplicity-controlled key secondary endpoints, including demonstrating significant reductions in triglycerides and apoC-III and in the incidence of acute pancreatitis in the pooled dose groups. In PALISADE, 25 mg REDEMPLO reduced triglycerides by a median of 80% from baseline versus a 17% reduction with placebo. Moreover, the odds of acute pancreatitis were 83% lower in the pooled dose groups (combined doses of 25 mg and 50 mg plozasiran) when compared with the placebo group (2 events in 2 subjects (4%) vs. 7 events in 5 subjects (20%), respectively). REDEMPLO is self-administered via subcutaneous injection once every three months.^1,2

The Australian Product Information notes the most common adverse reaction is hyperglycaemia (12.8%). Other common adverse reactions include headache (6.8%), nausea (4.7%), and injection site reaction (4.7%).

“Patients with FCS face a substantial burden of disease and are at life-long risk of acute pancreatitis, with few effective treatment options available,” said Gerald F Watts, DSc, PhD, MD, FRCP, Professor of Cardio-metabolic Medicine, University of Western Australia. “The results from the PALISADE study demonstrate that plozasiran can achieve substantial and sustained reductions in triglycerides in patients with FCS. These data highlight the potential of targeted RNA interference approaches in addressing conditions like FCS that have so far been difficult to treat. Plozasiran is a major advance in the care of FCS and a significant step toward expanding treatment options for patients.”