Arrowhead Pharmaceuticals’ REDEMPLO Gains NICE Recommendation for NHS Treatment of Familial Chylomicronaemia Syndrome in England and Wales
Arrowhead Pharmaceuticals’ REDEMPLO® (plozasiran) has received a positive funding recommendation from the National Institute for Health and Care Excellence (NICE), creating a new treatment option for eligible adults living with familial chylomicronaemia syndrome (FCS) in England and Wales. The therapy is intended to help reduce dangerously elevated triglyceride levels in patients whose condition has not been adequately controlled through diet and conventional triglyceride-lowering treatments.
Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) has announced that adults living with familial chylomicronaemia syndrome (FCS), a rare metabolic disorder associated with extremely high levels of triglycerides in the bloodstream, will have access to REDEMPLO® (plozasiran) through the National Health Service (NHS) in England and Wales, subject to the eligibility criteria outlined in the NICE guidance.
The decision represents an important development for people affected by FCS, a lifelong condition that can require strict dietary restrictions and carries a substantial risk of recurrent episodes of acute pancreatitis. These episodes can be painful, potentially life-threatening and may require hospitalisation.
REDEMPLO has received marketing authorisation in the United Kingdom as an adjunct to diet for reducing triglyceride levels in adult patients with FCS. The authorised indication allows patients to be diagnosed through genetic testing or clinical criteria. However, the NICE Technology Appraisal guidance specifically recommends NHS funding in England and Wales for adults with genetically confirmed FCS whose response to diet and conventional triglyceride-lowering therapies has been inadequate, subject to a commercial arrangement.¹˒²
The recommendation provides eligible patients with an additional treatment option alongside dietary management and existing therapies. It also highlights the importance of identifying FCS early and establishing an accurate diagnosis so that appropriate treatment can be considered.
NICE Recommendation Expands Treatment Options for Eligible Patients
FCS is an uncommon genetic metabolic disorder characterised by the body’s inability to process triglycerides effectively. Triglycerides are a type of fat circulating in the bloodstream that the body uses for energy. Although triglycerides are essential for normal physiological functions, extremely high concentrations can increase the risk of serious complications, particularly acute pancreatitis.
People living with FCS may experience persistently elevated triglyceride levels despite following a very-low-fat diet and receiving conventional lipid-lowering treatment. Because the condition is rare and its symptoms can overlap with those of other medical conditions, diagnosis may be delayed, leaving patients at risk of complications for extended periods.
The NICE recommendation is intended to address an important treatment need among adults with genetically confirmed FCS who continue to have inadequate disease control despite conventional management.
Under the recommendation, REDEMPLO can be used as an NHS-funded treatment option in England and Wales for patients who meet the specified eligibility requirements. The commercial arrangement associated with the recommendation forms part of the conditions governing NHS funding.
The regulatory and reimbursement decisions serve different purposes. The UK marketing authorisation establishes the approved use of REDEMPLO, while the NICE Technology Appraisal evaluates whether the treatment should be funded by the NHS for eligible patients in England and Wales.
The marketing authorisation covers the United Kingdom. However, the reimbursement process differs across the UK’s healthcare systems. Northern Ireland has an additional process for validating NICE guidance, while the Scottish Medicines Consortium separately considers NHS reimbursement in Scotland.
Consequently, the NICE recommendation applies formally to NHS care in England and Wales, and access arrangements in other parts of the United Kingdom are subject to their respective procedures.
Specialist Highlights the Importance of Early Diagnosis
Kausik Ray, Professor of Public Health at Imperial College London and Immediate Past President of the European Atherosclerosis Society, welcomed the introduction of another treatment option for people living with FCS.
Ray emphasised that the condition can expose patients to a lifelong risk of recurrent acute pancreatitis, which may be fatal in severe cases. He also highlighted the possibility that FCS can remain undiagnosed for years, preventing patients from receiving appropriate management.
The availability of REDEMPLO gives physicians an additional option when treating patients whose triglyceride levels remain inadequately controlled through diet and conventional therapies.
Ray pointed to results from the Phase 3 PALISADE study, which demonstrated a statistically significant reduction in median fasting triglyceride levels among patients receiving the recommended 25 mg dose of plozasiran.
At Month 10, the reduction was statistically significant compared with placebo, with a reported p-value of less than 0.0001.¹
These findings provide clinical evidence supporting the use of plozasiran to reduce triglyceride levels in adults with FCS.
Ray also underscored the need for physicians to recognise symptom patterns that may indicate the condition and consider genetic testing earlier when FCS is suspected.
Early identification can help distinguish FCS from other causes of severe hypertriglyceridaemia and support more appropriate treatment decisions. For patients who have experienced recurrent pancreatitis or continue to have extremely high triglyceride levels despite conventional management, timely assessment may be particularly important.
Patient Advocacy Group Welcomes the Decision
Action FCS, a UK charity focused on raising awareness and educating the public and healthcare professionals about familial chylomicronaemia syndrome, also welcomed the NICE recommendation.
Jill Prawer, Chair of Action FCS, highlighted the significant impact that FCS can have on daily life, particularly because managing the condition often requires an extremely fat-restricted diet.
For people living with FCS, dietary management is not limited to occasional adjustments. It can influence everyday decisions involving food preparation, shopping, eating outside the home and participating in social activities. Patients must remain attentive to dietary requirements to help manage triglyceride levels and reduce the risk of complications.
The burden can extend beyond dietary restrictions. The possibility of an acute pancreatitis episode may create ongoing uncertainty for patients and their families. Severe attacks can result in substantial pain, hospital admissions and interruptions to education, employment and family responsibilities.
Prawer noted that the availability of REDEMPLO through the NHS, alongside continued dietary management, offers renewed hope to patients and those who care for them.
The recommendation represents a potential improvement in the treatment options available to eligible adults, particularly those who have not achieved adequate control with conventional approaches.
Patient advocacy organisations play an important role in raising awareness of rare diseases such as FCS, supporting affected families and encouraging timely diagnosis. Greater awareness among healthcare professionals may also help reduce diagnostic delays and ensure that patients are evaluated for appropriate treatment options.
Arrowhead Emphasises Commitment to the UK FCS Community
Arrowhead Pharmaceuticals welcomed the NICE decision, describing the recommendation as an important step towards making plozasiran available to eligible patients through the NHS.
Graham Skarnvad, General Manager and Head of Europe at Arrowhead Pharmaceuticals, said the company was pleased to bring REDEMPLO to people living with FCS in England and Wales following the treatment’s approval and NHS reimbursement recommendation.
He also emphasised the company’s commitment to supporting the UK FCS community and expanding access to plozasiran in additional European countries over the coming months.
The announcement reflects Arrowhead’s efforts to advance the availability of its RNA interference-based medicines for patients with serious diseases. For people living with rare metabolic disorders, regulatory approval is an important milestone, but access also depends on reimbursement decisions and the implementation of healthcare-system funding arrangements.
The NICE recommendation provides a defined pathway for NHS-funded treatment in England and Wales for eligible adults with genetically confirmed FCS who have not responded adequately to diet and conventional triglyceride-lowering therapies.
Arrowhead’s stated plans to pursue availability in other European countries will depend on the regulatory, reimbursement and market-access requirements applicable in each jurisdiction.
REDEMPLO’s Regulatory Approval Supported by Phase 3 PALISADE Results
The UK marketing authorisation for REDEMPLO was supported by findings from the PALISADE clinical study, registered on ClinicalTrials.gov under identifier NCT05089084.
PALISADE was a Phase 3, placebo-controlled study designed to evaluate the efficacy and safety of plozasiran in 75 adults with FCS. Participants had either genetically confirmed disease or a clinical diagnosis of the condition.
The trial was designed to assess whether plozasiran could reduce fasting triglyceride levels in this patient population compared with placebo.
The primary endpoint was the median percentage change from baseline in fasting triglyceride levels at Month 10, comparing patients who received plozasiran with those who received placebo.¹
The study’s results showed that plozasiran produced a statistically significant reduction in median fasting triglyceride levels at the recommended 25 mg dose. The difference compared with placebo at Month 10 was highly statistically significant, with a p-value below 0.0001.
The result supports the potential of plozasiran to address one of the central metabolic abnormalities associated with FCS: persistently elevated triglyceride levels.
Because the condition is rare, clinical evidence from studies involving patients with FCS is particularly important when evaluating new therapeutic approaches. PALISADE included adults with genetically confirmed or clinically diagnosed FCS, allowing investigators to assess treatment in a population affected by this uncommon disorder.
Further details regarding the study design, eligibility criteria and clinical outcomes are available through the ClinicalTrials.gov record for PALISADE, study NCT05089084.
Although the reported triglyceride reduction is an important clinical finding, the study’s primary endpoint should be distinguished from direct evidence of a reduction in pancreatitis events. The reported results establish a significant effect on fasting triglyceride levels; the information provided in the announcement does not quantify a corresponding reduction in the frequency of acute pancreatitis episodes.
Understanding the Significance of Triglyceride Reduction in FCS
Triglyceride management is central to the care of people living with FCS because the condition can result in exceptionally high circulating triglyceride concentrations. These levels can increase the risk of acute pancreatitis, a serious inflammatory condition affecting the pancreas.
Standard management generally places substantial emphasis on dietary fat restriction and other conventional measures intended to reduce triglyceride levels. Nevertheless, some patients continue to experience inadequate control despite adhering to recommended approaches.
For these individuals, an additional treatment option may help address an important unmet medical need.
REDEMPLO is authorised for use as an adjunct to diet, meaning it is intended to complement rather than replace dietary management. The NICE recommendation similarly positions the therapy within the existing treatment framework for eligible adults who have not achieved adequate results with conventional approaches.
Treatment decisions will need to account for each patient’s diagnosis, clinical circumstances, previous response to treatment and the applicable prescribing guidance.
The availability of an additional therapy may also help reinforce the importance of specialist involvement in managing FCS. Healthcare professionals with experience in lipid disorders and rare metabolic diseases can help assess suspected cases, arrange appropriate diagnostic testing and determine whether patients meet the relevant treatment criteria.
Outlook for Patients and Healthcare Providers
The NICE recommendation marks a significant access milestone for adults with genetically confirmed FCS in England and Wales who continue to have inadequate triglyceride control despite diet and conventional triglyceride-lowering treatments.
By recommending NHS funding for REDEMPLO, NICE has established a pathway through which eligible patients can receive plozasiran under the specified commercial arrangement. The decision follows the therapy’s UK marketing authorisation and is supported by the statistically significant triglyceride reductions observed in the Phase 3 PALISADE study.
For patients and families, the development offers an additional option in managing a lifelong condition that can impose substantial dietary, physical and emotional burdens. For clinicians, it highlights the importance of recognising the signs of FCS, pursuing timely genetic testing where appropriate and considering available treatments when conventional management proves insufficient.
Arrowhead Pharmaceuticals intends to continue working towards broader availability of plozasiran in Europe. Meanwhile, the precise scope of access in the United Kingdom will continue to depend on the relevant national reimbursement processes and eligibility requirements.
The NICE recommendation does not eliminate the need for strict dietary management or ongoing medical supervision. However, it provides eligible adults in England and Wales with another treatment option aimed at reducing triglyceride levels and addressing a key feature of familial chylomicronaemia syndrome.
As awareness of FCS increases and additional treatment options become available, earlier diagnosis and appropriate access to specialist care will remain important priorities for improving the management of this rare metabolic disease.
About FCS
Familial Chylomicronemia Syndrome (FCS) is a severe and rare disease leading to extremely high triglyceride (TG) levels, typically over 10 mmol/L (880 mg/dL). Such severe elevations can lead to various serious signs and symptoms including acute and potentially fatal pancreatitis and other long-term complications.3
For more information about FCS, journalists can request a media backgrounder from Andrew.Smith@havas.com.
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals (NASDAQ: ARWR) is a commercial-stage pharmaceutical company developing medicines that treat intractable diseases by silencing the genes that cause them, harnessing the natural RNA interference (RNAi) mechanism. The company has built a portfolio of clinical and commercial RNAi therapeutics through its targeted RNAi molecule (TRiM™) platform. At Arrowhead, we rapidly advance potential RNAi treatments for diseases with significant unmet medical need, because every day matters to the patients we care for.