Pasadena, California, United States, October 27, 2026
Arrowhead Pharmaceuticals has announced a significant regulatory and healthcare access milestone for REDEMPLO® (plozasiran), offering a new treatment option for adults living with Familial Chylomicronaemia Syndrome (FCS) in England and Wales. The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation for REDEMPLO as an adjunct to diet to reduce triglyceride levels in adults diagnosed with FCS through genetic testing or clinical criteria. In addition, the National Institute for Health and Care Excellence (NICE) has issued guidance recommending NHS funding for the treatment in adults with genetically confirmed FCS, subject to a commercial arrangement. The development represents an important advancement for patients affected by this rare and potentially life-threatening metabolic disorder, which is associated with extremely elevated triglyceride levels and an increased risk of acute pancreatitis. The regulatory approval and NHS funding recommendation could improve access to an additional treatment option for eligible patients whose condition remains inadequately controlled through dietary measures and conventional triglyceride-lowering therapies.
MHRA Approval and NICE Recommendation Expand Treatment Access
The MHRA marketing authorisation allows REDEMPLO to be used in the United Kingdom as an additional treatment alongside dietary management for adults with FCS. Separately, NICE has recommended funding through the NHS in England and Wales for genetically confirmed adult patients whose response to diet and conventional triglyceride-lowering treatments has been inadequate. The recommendation is subject to an agreed commercial arrangement. This distinction is important because marketing authorisation establishes the approved use of a medicine, while NICE guidance addresses its availability through NHS-funded healthcare. The marketing authorisation covers the United Kingdom, whereas the NICE recommendation formally applies to England and Wales. Northern Ireland follows an additional process for validating NICE guidance, and reimbursement decisions in Scotland are considered separately by the Scottish Medicines Consortium. According to Arrowhead Pharmaceuticals, the company is working to expand access to plozasiran across additional European countries. The combined regulatory and reimbursement developments represent an important step toward making an RNA interference-based treatment available to eligible adults with this rare condition.
REDEMPLO Targets Extremely High Triglyceride Levels
Familial Chylomicronaemia Syndrome is a rare inherited metabolic disease characterised by severely elevated triglyceride concentrations in the bloodstream. Triglyceride levels in affected individuals can typically exceed 10 mmol/L (880 mg/dL), significantly increasing the risk of acute pancreatitis and other long-term complications. Patients may experience recurrent episodes of severe abdominal pain, hospitalisation and substantial lifestyle restrictions. Managing FCS often requires following an extremely restrictive, low-fat diet, which can affect daily activities, food choices and overall quality of life. Conventional triglyceride-lowering therapies may also provide inadequate control for some patients. REDEMPLO contains plozasiran, a small interfering RNA (siRNA) therapeutic designed to reduce the production of apolipoprotein C-III (APOC3), a protein involved in regulating triglyceride metabolism. By targeting APOC3 production, the treatment is intended to lower circulating triglyceride levels. It is administered as an additional treatment to dietary management rather than as a replacement for the recommended dietary restrictions. The availability of this therapeutic approach provides another option for addressing the underlying metabolic processes associated with FCS.
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Phase 3 PALISADE Study Supported Regulatory Approval
The marketing authorisation was supported by findings from the Phase 3 PALISADE clinical trial (NCT05089084), a placebo-controlled study evaluating the efficacy and safety of plozasiran in 75 adults with genetically confirmed or clinically diagnosed FCS. The primary endpoint assessed the median percentage change from baseline in fasting triglyceride levels compared with placebo at Month 10. According to the company, the recommended 25 mg dose demonstrated a statistically significant reduction in median fasting triglyceride levels compared with placebo at Month 10 (p<0.0001). These clinical findings provided important evidence supporting the treatment’s regulatory evaluation. The study also contributed to the growing clinical evidence for RNA interference-based medicines in rare metabolic diseases. Arrowhead Pharmaceuticals is a commercial-stage biotechnology company developing medicines through its targeted RNAi molecule (TRiM™) platform, which uses the body’s natural RNA interference mechanism to silence disease-associated genes. The latest UK development strengthens the company’s commercial position in the rare disease treatment landscape. With MHRA authorisation and NICE’s NHS funding recommendation, REDEMPLO represents an important new treatment option for eligible adults with FCS in England and Wales. Continued clinical experience and real-world treatment data will help further establish its role in long-term disease management.
Source: Arrowhead Pharmaceuticals press release
