PASADENA, Calif. — Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that adults in England and Wales who live with a rare metabolic condition called Familial Chylomicronaemia Syndrome (FCS) will have access to a newly licensed new treatment option available on the NHS to reduce extremely high levels of triglycerides (a type of fat) in their bloodstreams, if the response to diet and conventional triglyceride lowering therapies has been inadequate. REDEMPLO® (plozasiran) has received Marketing Authorisation as an adjunct to diet to reduce triglyceride levels in adult patients with FCS, diagnosed via genetic testing or clinical criteria.1 Following this marketing authorisation, NICE has issued Technology Appraisal guidance recommending funding for the NHS in England and Wales to use it as a treatment option to treat genetically confirmed FCS in adults if the response to diet and conventional triglyceride lowering therapies has been inadequate, in accordance with a commercial arrangement.2
“People living with FCS face a life-long risk of recurrent and potentially fatal acute pancreatitis that might remain undiagnosed for years, and physicians now have an important new option to treat this debilitating disease,” said Kausik Ray, Professor of Public Health at Imperial College, London and Immediate Past President of the European Atherosclerosis Society. “Results from the PALISADE study demonstrate that plozasiran significantly reduced median fasting triglyceride levels at the 25 mg recommended dose (p <0.0001 vs placebo) at month 10.1 By remaining aware of the combination of symptoms that suggest FCS, physicians can move to genetic testing earlier to confirm their diagnosis.”
“We warmly welcome today’s decision from NICE. Due to the need for an extremely fat-restricted diet, living with FCS affects every area of life, and every meal eaten. Alongside this can come the worry of a painful and debilitating attack of pancreatitis that can lead to time in hospital – time away from family, education and work. The availability of this new treatment option on the NHS, alongside the diet, brings real hope to people living with FCS and those who care for them,” added Jill Prawer, Chair of Action FCS, a UK charity dedicated to raising awareness and educating about FCS.
Marketing authorisation for REDEMPLO covers the United Kingdom, while the NICE guidance formally applies to the NHS in England and Wales. Northern Ireland has an additional process to validate NICE guidance, while NHS reimbursement in Scotland is considered separately by the Scottish Medicines Consortium.
“We are thrilled to bring REDEMPLO as a new treatment option, approved and reimbursed on the NHS in England for people living with FCS,” said Graham Skarnvad, General Manager and Head of Europe at Arrowhead Pharmaceuticals. “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.”
Regulatory approval for REDEMPLO was based on findings from the PALISADE study (NCT05089084), a Phase 3 placebo-controlled study to evaluate the efficacy and safety of plozasiran in 75 adults with genetically confirmed or clinically diagnosed FCS. The primary endpoint of the study was the median percent change from baseline in fasting TGs versus placebo at Month 10.4 Additional information on study design can be found on clinicaltrials.gov (NCT05089084).
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 [email protected].
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.
For more information, please visit https://arrowheadpharma.com/en-us or follow our U.K. channel on LinkedIn. Journalists can request a media backgrounder from [email protected].
References
- Redemplo (plozasiran) Summary of Product Characteristics, U.K. Arrowhead Pharmaceuticals Ireland Limited; October 2026.
- National Institute for Health and Care Excellence, Technology Appraisal Guidance ID6593: Plozasiran for treating familial chylomicronaemia syndrome, available via https://www.nice.org.uk/guidance/indevelopment/gid-ta11793 [Accessed October 2026].
- Goldberg RB, Chait A, et al. Front Endocrinol (Lausanne). 2020;11:593931.
- Watts GF, Rosenson RS, Hegele RA, Goldberg IJ, Gallo A, Mertens A, Baass A, Zhou R, Muhsin M, Hellawell J, et al. Plozasiran for managing persistent chylomicronemia and pancreatitis risk.
N Engl J Med. 2024;392:127–137. https://doi.org/10.1056/nejmoa2409368 PMID: 39225259.
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