Arrowhead Pharmaceuticals has received approval from the Australian Therapeutic Goods Administration (TGA) for Redemplo (plozasiran) to treat familial chylomicronemia syndrome (FCS).
The therapy is approved as an add-on to diet, specifically for adults with FCS whose triglyceride levels remain high despite standard triglyceride-lowering treatments.
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The approval marks an expansion of access for a genetic disorder estimated to affect one to 13 people per million worldwide.
Redemplo is said to be the only medicine currently approved in Australia for the treatment of FCS in adults, covering both genetically confirmed and clinically diagnosed cases.
It adds to previous authorisations in Canada, China, and the US, as well as a positive opinion for marketing authorisation in the European Union.
Redemplo utilises Arrowhead’s TRiM platform and is designed to silence the messenger ribonucleic acid (mRNA) encoding apolipoprotein C-III (apoC-III), a regulator of triglyceride metabolism.
FCS results in high triglyceride levels, increasing the risk of recurrent and potentially fatal pancreatitis.
The TGA approval is backed by data from the Phase III PALISADE trial, a double-blind, placebo-controlled, randomised study conducted across 39 sites worldwide, including five in Australia.
PALISADE met its primary and all controlled key secondary endpoints, with Redemplo 25mg reducing triglycerides by a median of 80% from baseline, compared to 17% with placebo.
The risk of acute pancreatitis was 83% lower in pooled dose groups relative to placebo.
Redemplo is administered as a subcutaneous injection every three months. The most common reported adverse reaction was hyperglycaemia (12.8%), with headache, nausea, and injection site reactions also observed.
Efficacy and safety results from the PALISADE study have been presented at the European Society of Cardiology Congress 2024 and American Heart Association Scientific Sessions 2024, and published in The New England Journal of Medicine and Circulation.
Redemplo was reviewed by Australia’s TGA under the priority review pathway and has also received breakthrough therapy, fast track, and orphan drug designations in the US, as well as orphan medicinal product designation in the European Union.
Arrowhead Pharmaceuticals president and CEO Christopher Anzalone said: “We are pleased that Redemplo is now the first-ever approved treatment for Australians living with genetic or clinical FCS.
“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.”
