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 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.

For more information, please follow our U.K. channel on LinkedIn. Journalists can request a media backgrounder from Andrew.Smith@havas.com.

References

  1. Redemplo (plozasiran) Summary of Product Characteristics, U.K. Arrowhead Pharmaceuticals Ireland Limited; October 2026.

  2. 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].

  3. Goldberg RB, Chait A, et al. Front Endocrinol (Lausanne). 2020;11:593931.

  4. 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.

Safe Harbor Statement under the Private Securities Litigation Reform Act:

This news release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Any statements contained in this release except for historical information may be deemed to be forward-looking statements. Without limiting the generality of the foregoing, words such as “may,” “will,” “expect,” “believe,” “anticipate,” “hope,” “intend,” “plan,” “project,” “could,” “estimate,” “continue,” “target,” “forecast” or “continue” or the negative of these words or other variations thereof or comparable terminology are intended to identify such forward-looking statements. In addition, any statements that refer to projections of our future financial performance, trends in our business, expectations for our product pipeline, products or product candidates or other characterizations of future events or circumstances are forward-looking statements. These forward-looking statements include, but are not limited to, statements about our beliefs and expectations regarding the long-term impacts of REDEMPLO (plozasiran) on patient health and the health care system; our beliefs and expectations regarding the pricing, value, or expected timing for availability of our drugs and drug candidates if approved; and our beliefs and expectations around the potential uses and value of the TRiM™ platform. These statements are based upon our current expectations and speak only as of the date hereof. Actual results or outcomes may differ materially and adversely from those expressed in any forward-looking statements as a result of numerous factors and uncertainties, including the safety and efficacy of our products and product candidates, pricing and reimbursement decisions related to our products if approved, demand for our products, decisions of regulatory authorities and the timing thereof, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of our scientific studies, the timing for starting and completing clinical trials, rapid technological change in our markets, the enforcement of our intellectual property rights, and the other risks and uncertainties described in our most recent Annual Report on Form 10-K, subsequent Quarterly Reports on Form 10-Q and other documents filed with the Securities and Exchange Commission from time to time. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances.

Source: Arrowhead Pharmaceuticals, Inc.

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