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news.Arrowhead Pharmaceuticals has received marketing authorisation from the UK Medicines and Healthcare products Regulatory Agency (MHRA) for Redemplo (plozasiran) as an adjunct to diet to reduce triglyceride levels in adults with familial chylomicronaemia syndrome (FCS).
The regulatory clearance was supported by results from the PALISADE study. Credit: PeopleImages / Shutterstock.com.
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Concurrently, the National Institute for Health and Care Excellence (NICE) has published Technology Appraisal guidance recommending funding for the National Health Service (NHS) for adult patients with genetically confirmed disease.
The regulatory approval covers patients diagnosed through either genetic testing or clinical criteria.
NHS funding recommended by the NICE applies to eligible patients whose condition does not respond adequately to diet and standard triglyceride-lowering treatments, under an agreed commercial arrangement.
Coverage details vary across the devolved administrations, encompassing England, Northern Ireland, Scotland, and Wales.
The licensing clearance applies across the entire UK, whereas the health institute’s recommendation formally directs NHS care in England and Wales.
Northern Ireland maintains a separate administrative validation procedure for this guidance, and medicine reimbursement in Scotland remains subject to independent appraisal by the Scottish Medicines Consortium.
Arrowhead Pharmaceuticals general manager and Europe head Graham Skarnvad said: “We are thrilled to bring Redemplo as a new treatment option, approved and reimbursed on the NHS in England for people living with FCS.
“Supporting the FCS community in the UK is an important focus for us as we work to make plozasiran available for people living with FCS in several European countries over the coming months.”
The regulatory clearance was supported by results from the PALISADE study, a Phase III, placebo-controlled trial evaluating the safety and efficacy of plozasiran in 75 adult participants with clinically diagnosed or genetically confirmed FCS.
The primary measure in the trial was the median percentage change from baseline in fasting triglyceride concentrations relative to placebo at month ten.
FCS is a severe and rare metabolic disorder that triggers extreme elevations in blood triglycerides, typically reaching levels above 10mmol/L (880mg/dL).
In May, Arrowhead received approval from the Australian Therapeutic Goods Administration for Redemplo to treat FCS.
